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PANTHER Study of Terumo Aortic Knitted and Woven Grafts, and Cardiovascular Patches (PANTHER)
Clinical Study Manager - r.smith1@terumoaortic.com
NCT04545502
Inclusion Criteria:
* Patients must meet all of the following inclusion criteria in order to be eligible for inclusion in the study:
All Patients:
* Patient meets the minimum age as per local regulations at time of consent
* Patient requires treatment with study device(s) according to the IFU(s)
* Patient is willing and able to comply with all SOC procedures and study visits
* Patient or their legally authorised representative (LAR) has given written informed consent to participate in study, including consent to collect data retrospectively
* For prospective emergency patients, retrospective consent is permissible
* For retrospective patients who are deceased at time of enrollment, local EC regulations relating to consent process should be followed.
Retrospective Patients only:
* Patient has a minimum of 1 year post-operative follow-up data available, or complete data to death.
* Patient implant date is no more than 5 years prior to study start date.
Exclusion Criteria:
* Any patient who meets any of the following exclusion criteria will be excluded from participation in the study:
* Patient is unable or unwilling to comply with the SOC procedures or follow-up regime
* Patient is contraindicated per the device IFU
* Patient has any other medical, social or psychological problems that in the opinion of the investigator preclude them from receiving this treatment and the procedures and evaluations pre- and post-procedure DEVICE: Gelsoft Plus Vascular Graft, DEVICE: Vascular Bypass Graft, DEVICE: Cardiovascular Patch, DEVICE: Gelweave Vascular Graft, DEVICE: Gelweave Valsalva Vascular Graft
Aneurysm, Dissection, Aortic Root Aneurysm, Aortic Root Dissection, Carotid Artery Injuries, Aortic Arch, Bypass Extremity Graft, Occlusive Vascular Disease, Aortic Diseases
Vascular Surgery
Testing the Addition of Radiation Therapy to the Usual Immune Therapy Treatment (Atezolizumab) for Extensive Stage Small Cell Lung Cancer, The RAPTOR Trial (RAPTOR)
Hamilton, Melanie, R - mrhamilton2@vcu.edu
Weiss, Elisabeth
NCT04402788
HM20020924
Inclusion Criteria:
* Any confirmation (cytologic, histologic, or pathologic) of extensive stage small cell lung cancer at any site, either primary or metastases
* Partial response (PR) or stable disease (SD) after 4-6 cycles of etoposide/platinum (E/P) doublet plus atezolizumab by re-staging scans (positron emission tomography \[PET\]/computed tomography \[CT\] scan, diagnostic CT scan, magnetic resonance imaging \[MRI\] optional per treating physician); atezolizumab should continue through randomization. Patients must be randomized within 9 weeks of last dose of etoposide/platinum (if not receiving PCI) or 6 weeks from completion of prophylactic cranial irradiation (PCI)
* NOTE: Patients must have at least 3 cycles of E/P plus atezolizumab. They can have one cycle of induction E/P without concurrent atezolizumab if unable to receive concurrent E/P combined with atezolizumab for all cycles of induction therapy
* Patients must have measurable disease (per Response Evaluation Criteria in Solid Tumors \[RECIST\]) and 3 or fewer observable liver metastases and no evidence of progressive disease (per RECIST) at time of enrollment
* At time of enrollment after induction E/P chemotherapy and atezolizumab, if there is a pleural effusion, patients will be eligible if thoracentesis is cytologically negative or if pleural fluid is too small a volume to effectively sample by thoracentesis and does not show increased metabolic activity on CT/PET imaging
* Appropriate stage for study entry based on the following diagnostic workup:
* History/physical examination within 14 days prior to registration;
* Imaging within 42 days prior to registration to include:
* MRI brain with contrast or CT brain with contrast
* CT chest, abdomen and pelvis or whole body PET/CT scan any time after the fourth cycle of chemotherapy and prior to registration
* Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 within 14 days prior to registration
* Absolute neutrophil count (ANC) \>= 1,000/cells/mm\^3 (within 14 days prior to registration)
* Platelets \>= 75,000 cells/mm\^3 (within 14 days prior to registration)
* Hemoglobin \>= 8 g/dL (within 14 days prior to registration)
* Total bilirubin =\< 1.5 x upper limit of normal (ULN) (within 14 days prior to registration)
* Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 3.0 x ULN (AST and/or ALT =\< 5 ULN for patients with liver involvement) (within 14 days prior to registration)
* Alkaline phosphatase =\< 2.5 x ULN (=\< 5 ULN for patients with documented liver involvement or bone metastases) (within 14 days prior to registration)
* Adequate renal function = Creatinine clearance \>=40 mL/min by the Cockcroft-Gault (C-G) equation: (within 14 days prior to registration)
* Upfront radiation therapy of symptomatic metastatic site is permissible if causing symptoms such as pain or impending fracture
* Patients with brain metastases are eligible after receiving whole brain radiation before enrollment (anytime during induction systemic therapy). Whole brain radiation can be delivered with hippocampal sparing or 3-D conformal technique. Patients with irradiated brain metastases are eligible if they are clinically stable from a neurological standpoint after completing radiotherapy (e.g. not having uncontrolled seizures) and do not require use of steroids above a dose of 10 mg of prednisone daily
* For women of childbearing potential, a negative serum or urine pregnancy test within 14 days prior to registration.
* Note: Women will be considered post-menopausal if they have been amenorrheic for 12 months without an alternative medical cause. The following age-specific requirements apply:
* Women \< 50 years of age would be considered post-menopausal if they have been amenorrheic for 12 months or more following cessation of exogenous hormonal treatments and if they have luteinizing hormone and follicle-stimulating hormone levels in the post-menopausal range for the institution or underwent surgical sterilization (bilateral oophorectomy or hysterectomy)
* Women \>= 50 years of age would be considered post-menopausal if they have been amenorrheic for 12 months or more following cessation of all exogenous hormonal treatments, had radiation-induced menopause with last menses \> 1 year ago, had chemotherapy-induced menopause with last menses \> 1 year ago, or underwent surgical sterilization (bilateral oophorectomy, bilateral salpingectomy or hysterectomy)
* Patients positive for human immunodeficiency virus (HIV) on effective anti-retroviral therapy with undetectable viral load within 6 months and a stable regimen of highly active anti-retroviral (HAART) HIV-positive patients must have no requirement for concurrent antibiotics or antifungal agents for the prevention of opportunistic infections
* The patient or a legally authorized representative must provide study-specific informed consent prior to study entry
Exclusion Criteria:
* Metastatic disease invading the liver (\> 3 metastases), heart or \> 10 metastatic sites detectable after induction systemic therapy. Each visible bone metastasis on radiographic scan counts as one site
* Patients with a concurrent malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessment of the investigational regimen with atezolizumab or radiation
* Prior radiotherapy in the thorax that would result in overlapping RT fields, unless the overlapping fields meet acceptable dose constraints for normal tissue
* Active autoimmune disease, including, but not limited to: systemic lupus erythematosus; rheumatoid arthritis; inflammatory bowel disease (e.g. Crohn's, ulcerative colitis); vascular thrombosis associated with antiphospholipid syndrome; Wegener's granulomatosis; Sjogren's syndrome; Guillain-Barre syndrome; multiple sclerosis; vasculitis; or glomerulonephritis.
* If the autoimmune disease is not active for over 3 years and the patient is not receiving immunosuppressive treatment such as methotrexate or steroids above a dose equivalent to 10 mg prednisone daily, the patient is eligible.
* Patients with a history of autoimmune hypothyroidism on a stable dose of thyroid replacement hormone are eligible
* Patients with controlled type 1 diabetes mellitus on a stable insulin regimen are eligible
* Patients with eczema, psoriasis, lichen simplex chronicus or vitiligo with dermatologic manifestations are excluded only if they have active disease with acute exacerbation and on immunosuppressive medications within the 12 months prior to enrollment. They are eligible otherwise.
* Severe, active co-morbidity defined as follows:
* Any other diseases, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates the use of an investigational drug or that may affect the interpretation of the results or render the patient at high risk from treatment complications;
* Active tuberculosis;
* Known clinically significant liver disease, including active viral, alcoholic, or other hepatitis; cirrhosis; fatty liver; and inherited liver disease
* Patients with past or resolved hepatitis B infection (defined as having a negative hepatitis B surface antigen \[HBsAg\] test and a positive anti-HBc \[antibody to hepatitis B core antigen\] antibody test) are eligible
* Patients positive for hepatitis C virus (HCV) antibody are eligible only if polymerase chain reaction (PCR) is negative for HCV ribonucleic acid (RNA). (The HCV RNA test must be performed for patients who have a positive HCV antibody test)
* Known immunosuppressive disease, for example history of bone marrow transplant or chronic lymphocytic leukemia (CLL);
* Chronic obstructive pulmonary disease (COPD) requiring chronic oral steroid therapy of \> 10 mg prednisone daily or equivalent at the time of registration. Inhaled corticosteroids are not exclusionary;
* Unstable angina and/or congestive heart failure requiring hospitalization within the last 3 months;
* History of recent myocardial infarction within 6 months prior to registration.
* Clinically significant interstitial lung disease
* Pregnancy: Administration of atezolizumab may have an adverse effect on pregnancy and poses a risk to the human fetus, including embryo-lethality. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study treatment, and for 5 months (150 days) after the last dose of study agent. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately
* Women who are breastfeeding and unwilling to discontinue
* History of allogeneic organ transplant
* Patients who have had immunotherapy-induced pneumonitis DRUG: Atezolizumab, PROCEDURE: Biospecimen Collection, PROCEDURE: Computed Tomography, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Positron Emission Tomography, RADIATION: Radiation Therapy
Extensive Stage Lung Small Cell Carcinoma
Evaluating the Safety and Efficacy of Polyethylene Glycol (PEG) Mediated Fusion (PEG Fusion)
Elizabeth Wysocki, MA - ewysock2@jhu.edu
NCT04789044
Inclusion Criteria:
• Ages 18-80
• Sustained a complete peripheral nerve injury resulting from upper extremity trauma presenting within 48 hours of injury
• Involves injury or dysfunction to motor and/or sensory function of the axillary, radial, median, ulnar, or musculocutaneous nerve with injury localized between the proximal humerus and the distal flexion crease of the wrist
• Involves a "mixed" nerve segment (i.e., involves a location of any of the above specified nerves that can be expected to have both sensory and motor axons present).
Exclusion Criteria:
• Patients beginning surgery within hours after injury.
• Injury to the brachial plexus nerves
• Injury to the nerves distal to the distal flexion crease of the wrist
• Injury that involves a distal extension of the parent nerve that is considered sensory only (superficial radial nerve, lateral antebrachial cutaneous nerve, etc.) or the posterior interosseus or anterior interosseus nerve distal to the midpoint of the forearm (i.e., distal to what can be considered the main motor branches of the PIN and AIN).
• Previous peripheral nerve injury resulting from trauma, stroke, muscular, neurologic, or neuromuscular disorder
• Documented psychiatric disorder that is expected to result in high probability of self-harm or interfere with study follow-up.
• Severe problems with maintaining follow up (e.g., patients who are prisoners or homeless at time of injury or who are intellectually challenged without adequate family support).
• Not expected to survive the next 30 days due to their injuries/health condition.
• The subject has a known allergy to polyethylene glycol (PEG).
• If any of the assessments cannot be done on the contralateral side (CL) or the MRCC sensory 2PD value is \> 10 mm on the CL side during baseline period, the subject is a screen failure.
• The subject is pregnant and/or is breastfeeding.
• The subject has a significant medical comorbidity precluding immediate repair.
• The subject is not able to strictly adhere to the rules of the current clinical protocol.
DRUG: NTX-001
Peripheral Nerve Injuries
A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations
Gwaltney, Lindsey - lbgwaltney@vcu.edu
Griffin, Jordyn
NCT04293562
HM20020887
Inclusion Criteria:
* All patients must be enrolled on APEC14B1 and consented to Eligibility Screening (Part A) prior to enrollment and treatment on AAML1831
* Patients must be less than 22 years of age at the time of study enrollment
* Patient must be newly diagnosed with de novo AML according to the 2016 World Health Organization (WHO) classification with or without extramedullary disease
* Patient must have 1 of the following:
* \>= 20% bone marrow blasts (obtained within 14 days prior to enrollment)
* In cases where extensive fibrosis may result in a dry tap, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy
* \< 20% bone marrow blasts with one or more of the genetic abnormalities associated with childhood/young adult AML as provided in the protocol (sample obtained within 14 days prior to enrollment)
* A complete blood count (CBC) documenting the presence of at least 1,000/uL (i.e., a white blood cell \[WBC\] count \>= 10,000/uL with \>= 10% blasts or a WBC count of \>= 5,000/uL with \>= 20% blasts) circulating leukemic cells (blasts) if a bone marrow aspirate or biopsy cannot be performed (performed within 7 days prior to enrollment)
* ARM C: Patient must be \>= 2 years of age at the time of Late Callback
* ARM C: Patient must have FLT3/ITD allelic ratio \> 0.1 as reported by Molecular Oncology
* ARM C: Patient does not have any congenital long QT syndrome or congenital heart block
* ARM C: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
* ARM C: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
* ARM C: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
* ARM D: Patient must be \>= 2 years of age at the time of Late Callback
* ARM D: Patient must have one of the clinically relevant non-ITD FLT3 activating mutations as reported by Foundation Medicine
* ARM D: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
* ARM D: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
* ARM D: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
* NEUROPSYCHOLOGICAL TESTING: Patient must be enrolled on Arm A or Arm B. Patients who transfer to Arm C or Arm D are not eligible
* NEUROPSYCHOLOGICAL TESTING: Patient must be 5 years or older at the time of enrollment
* NEUROPSYCHOLOGICAL TESTING: English-, French- or Spanish-speaking
* NEUROPSYCHOLOGICAL TESTING: No known history of neurodevelopmental disorder prior to diagnosis of AML (e.g., Down syndrome, fragile X, William syndrome, mental retardation)
* NEUROPSYCHOLOGICAL TESTING: No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli
* All patients and/or their parents or legal guardians must sign a written informed consent
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:
* Fanconi anemia
* Shwachman Diamond syndrome
* Patients with constitutional trisomy 21 or with constitutional mosaicism of trisomy 21
* Telomere disorders
* Germline predispositions known, or suspected by the treating physician to increase risk of toxicity with AML therapy
* Any concurrent malignancy
* Juvenile myelomonocytic leukemia (JMML)
* Philadelphia chromosome positive AML
* Mixed phenotype acute leukemia
* Acute promyelocytic leukemia
* Acute myeloid leukemia arising from myelodysplasia
* Therapy-related myeloid neoplasms
* Patients with persistent cardiac dysfunction prior to enrollment, defined as ejection fraction (EF) \< 50% (preferred method Biplane Simpson's EF) or if EF unavailable, shortening fraction (SF) \< 24%. \*Note: if clinically safe and feasible, repeat echocardiogram is strongly advised in order to confirm cardiac dysfunction following clinical stabilization, particularly if occurring in the setting of sepsis or other transient physiologic stressor. If the repeat echocardiogram demonstrates an EF \>= 50%, the patient is eligible to enroll and may receive an anthracycline-containing Induction regimen
* Administration of prior anti-cancer therapy except as outlined below:
* Hydroxyurea
* All-trans retinoic acid (ATRA)
* Corticosteroids (any route)
* Intrathecal therapy given at diagnosis
* In particular, strong inducers of CYP3A4 and/or P-glycoprotein (P-gp) should be avoided from the time of enrollment until it is determined whether the patient will receive gilteritinib. Patients receiving gilteritinib will be required to avoid strong CYP3A4 inducers and/or strong P-gp inducers for the duration of the study treatment
* Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
* Lactating females who plan to breastfeed their infants
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
* ARM D: Patient does not have any congenital long QT syndrome or congenital heart block PROCEDURE: Allogeneic Hematopoietic Stem Cell Transplantation, DRUG: Asparaginase Erwinia chrysanthemi, PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Bone Marrow Biopsy, PROCEDURE: Computed Tomography, DRUG: Cytarabine, DRUG: Daunorubicin Hydrochloride, DRUG: Dexrazoxane Hydrochloride, DRUG: Etoposide, OTHER: Fludeoxyglucose F-18, DRUG: Gemtuzumab Ozogamicin, DRUG: Gilteritinib Fumarate, DRUG: Liposome-encapsulated Daunorubicin-Cytarabine, PROCEDURE: Magnetic Resonance Imaging, DRUG: Methotrexate, DRUG: Mitoxantrone Hydrochloride, PROCEDURE: Positron Emission Tomography, OTHER: Questionnaire Administration, DRUG: Therapeutic Hydrocortisone
Acute Myeloid Leukemia
Preventing Retaliatory Gun Violence in Violently Injured Adults
Nicholas Thomson - Nicholas.Thomson@vcuhealth.org
NCT04813185
Inclusion Criteria:
* They receive treatment in the hospital for a violence-related injury (e.g., gunshot wound)
* They are 18 years or older
* They are English speaking
* They are eligible for BTG services (which includes living within the BTG catchment area for the hospital; Richmond City and neighboring counties)
Exclusion Criteria:
* Non-English speaking
* Age \<18
* Prisoners BEHAVIORAL: Bridging the Gap
Violence
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
Reference Study ID Number: DSC/14/2357/51 - patientadvocacy@italfarmaco.com
Harper, Amy
NCT03373968
HM20018448
Inclusion Criteria:
• Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in study DSC/14/2357/48 and met: * all the inclusion criteria and none of the exclusion criteria, * had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or \>30%, i.e. included in"off-target" group, * never been randomized because, the enrollment in the off target group was completed.
• Aged ≥6 years old;
• Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to localregulations);
• Subjects must be willing to use adequate contraception: * Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following: * True abstinence (absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the subject. * Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception. * Condom with spermicide and the female partner must use an acceptable method of contraception, such as an oral, * transdermal, injectable or implanted steroid-basedcontraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such asfor example cervical cap with spermicide jelly.
Exclusion Criteria:
• Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed;
• Use of any current investigational drug other than Givinostat;
• Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results;
• Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD;
• Have platelets count, White Blood Cell and Hemoglobin at screening \< Lower Limit of Normal (LLN)\* (for abnormal screening laboratory test results (\
• Have inadequate renal function, as defined by serum Cystatin C \>2 x the upper limit of normal (ULN) at screening visit\*. If the value is \>2 x ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \>2 x ULN, the subject should be excluded);
• Have heart failure (New York Heart Association Class III or IV)
• Have a current liver disease or impairment, including but not limited to an elevated total bilirubin\* (i.e. \> 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's;
• Have a baseline QTcF \>450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome);
• Have a psychiatric illness/social situation rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures.
• Have any hypersensitivity to the components of study medication;
• Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance. * the Investigators to evaluate these exclusion criteria can use the laboratory results obtained within 5 months from V1, to allow the continuity of the treatment. It is worth noting, as soon as the site will receive the laboratory results done in screening/baseline (Visit 1) visit they will check the GIVINOSTAT dose and modify it as per protocol safety rules and/or dosage modifications rules.
DRUG: Givinostat
Duchenne Muscular Dystrophy
Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) (RESTORE)
Novartis Gene Therapies - novartis.email@novartis.com
Johnson, Nicholas, E
NCT04174157
HM20015046
Inclusion Criteria:
* Patients treated with OAV-101 with a genetically confirmed diagnosis of SMA regardless of the date of diagnosis.
* Appropriate consent/assent has been obtained for participation in the registry
Exclusion Criteria:
• Currently enrolled in an interventional clinical trial involving an investigational medicinal product to treat SMA. Note: Patients who are participating in a Compassionate Use Program (CUP) for OAV-101 (Zolgensma) such as a Managed Access Program (MAP), an Expanded Access Program (EAP), Single Patient Investigational New Drug (IND) (SPI) or Named Patient Program (NPP) are eligible to enroll in the registry regardless of the date of a genetic or clinical diagnosis of SMA.
OTHER: Prospective observational registry, DRUG: Zolgensma
Spinal Muscular Atrophy (SMA)
Post Approval Study for Treatment of Drug-resistant Adult and Pediatric Primary FSGS Using the LIPOSORBER® LA-15 System (FSGSALLAGE)
Ayaka Kitamura - Ayaka.Kitamura1@kaneka.co.jp
NCT04065438
Inclusion Criteria:
A patient is deemed suitable for inclusion in the study if the patient has nephrotic syndrome associated with primary FSGS when:
• Standard treatment options, including corticosteroid and/or calcineurin inhibitors, are unsuccessful or not well tolerated and the patient's glomerular filtration rate (GFR) ≥ 45 ml/min/1.73 m2.
or
• The patient is post renal transplantation.
Exclusion Criteria:
General Exclusion Criteria
• Patient is greater than 75 years of age at the start of the treatment period or less than 22
• The patient is unwilling or unable to sign and date the informed consent
• Pregnant, lactating, or planning to become pregnant prior to completing the study (Note: The safety of the use of LIPOSORBER® in pregnant women has not been studied. There may be unknown risks to an embryo/fetus. Sexually active women of childbearing potential should avoid pregnancy during the use of the LIPOSORBER device and throughout the study duration.)
• Unable or unwilling to comply with the follow-up schedule
• Simultaneously participating in another investigational drug or device study
• Body weight \< 15 kg (33.1 lbs) Medical Exclusion Criteria
• Currently being administered ACE inhibitors that cannot be withheld for at least 24 hours prior to each apheresis treatment (Note: The time period to withhold ACE inhibitors should be prolonged, if determined by the treating physician, considering each individual's renal function and the biological half-life of the ACE-inhibitor currently in use.)
• Currently being administered antihypertensive drugs other than ACE inhibitors (e.g., ARBs) that cannot be withheld on the day of apheresis until after the procedure
• Medical condition or disorder that would limit life expectancy to less than the primary clinical study endpoint or that may cause noncompliance with the study plan or confound the data analysis
• Hypersensitivity to dextran sulfate, heparin, or ethylene oxide
• Adequate anticoagulation cannot be achieved due to severe hemophilia, severe hemorrhage diathesis, severe gastrointestinal ulcers, or are recipients of vitamin K antagonist medications
• Extracorporeal circulation therapy with LIPOSORBER® LA-15 System cannot be tolerated due to severe cardiac insufficiency, acute myocardial infarction, severe cardiac arrhythmia, acute apoplexy, severe uncontrollable hypertension, or severe uncontrollable hypotension Note: Severe uncontrollable hypotension/hypertension indicates the cases with systolic and/or diastolic blood pressure ≤ 5th percentile for age, gender, and height.
• Cardiac impairments such as uncontrolled arrhythmia, unstable angina, decompensated congestive heart failure, or valvular disease
• Functional thyroid disease or liver abnormalities
• Unresolved systemic or local infection that could affect the clinical study outcomes
DEVICE: LIPOSORBER® LA-15
Focal Segmental Glomerulosclerosis
FSGS, Liposorber
Study to Test OBI-3424 in Patients With T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LBL)
Weir, Caryn, R - cweir@vcu.edu
Maher, Keri
NCT04315324
HM20021956
Inclusion Criteria:
* Patients must have a diagnosis of relapsed or refractory T-cell acute lymphoblastic leukemia (T-ALL) based on World Health Organization (WHO) classification. Patients with relapsed/refractory T-cell lymphoblastic lymphoma are eligible if lymphoblasts are \>= 5% in the bone marrow or in the peripheral blood by morphology or flow cytometry
* Patients must have evidence of acute leukemia in their peripheral blood or bone marrow. Patients must have \>= 5% lymphoblasts in the peripheral blood or bone marrow within 14 days prior to registration. Patients with only extramedullary disease are not eligible
* Patients ≥ 18 years of age must be refractory to or have relapsed following a standard induction chemotherapy. Patients \< 18 years of age must have relapsed or must be refractory after 2 or more chemotherapy cycles (example: induction and consolidation)
* A standard chemotherapy induction regimen is defined as any program of treatment that includes:
* Vincristine and corticosteroids plus at least one more chemotherapy agent
* Cytarabine and anthracycline, or
* High dose cytarabine (defined as at least 1 gr/m\^2 per individual dose unless adjustments were required for renal/liver function)
* Patients must have no evidence of central nervous system disease within 28 days prior to registration based on cerebrospinal fluid (CSF) studies. Patients with clinical signs or symptoms consistent with central nervous system (CNS) involvement must have a lumbar puncture which is negative for CNS involvement; the lumbar puncture must be completed within 28 days prior to registration. Patients with CNS1 or CNS2 are eligible; however patients with CNS3 are not eligible
* Note that the patients may receive intrathecal chemotherapy with the initial lumbar puncture. This may count as the first dose of intrathecal therapy required as part of the study
* Prior nelarabine therapy is not required. In addition, for patients ≥ 18 years of age who received nelarabine during initial induction or post-remission treatment are eligible only if the physician does not feel they would benefit from other, multi-agent chemotherapy
* Patients must not have had chemotherapy or investigational agents within 14 days prior to registration except for corticosteroids, oral 6-mercaptopurine, oral methotrexate, vincristine, intrathecal chemotherapy, or hydroxyurea. For participants who have received radiation therapy, at least 7 days must have elapsed from the end of radiation prior to registration and participants must not currently be experiencing toxicities from radiation therapy
* Patients must not have undergone allogeneic hematopoietic transplant within 90 days prior to registration
* Patients must have no evidence of active \>= grade 2 acute graft versus host disease (GVHD) or moderate or severe limited chronic GVHD. Patients must have no history of extensive GVHD of any severity within 90 days prior to registration. Patients who are post-transplant must be off calcineurin inhibitors for at least 21 days to be eligible. Extensive GVHD is defined as 1) generalized skin involvement or 2) localized skin involvement and/or hepatic dysfunction plus liver histology or cirrhosis or involvement of eye or minor salivary organ or oral mucosa or any other target organ
* Patients must be \>= 12 years of age
* Patients ≥ 16 years of age must have a Zubrod Performance Status of 0-3. Patients \< 16 years of age must have a Lansky score of ≥ 50
* Patients must not have systemic fungal, bacterial, viral or other infection that is not controlled (defined as exhibiting ongoing signs/symptoms related to the infection and without improvement, despite appropriate antibiotics or other treatment) within 14 days prior to registration
* Patients ≥ 18 years of age must have creatinine clearance \> 30 mL/min within 14 days prior to registration according to the Cockcroft Gault equation
* Patients 12-17 years of age must have adequate renal function within 14 days prior to registration defined as serum creatinine ≤ 1.5 x institutional upper limit of normal (ULN) according to age or a calculated estimated glomerular filtration rate (eGFR) (based on Schwartz formula) or radioisotope glomerular filtration rate (GFR) ≥ 50ml/min/1.73 m\^2
* Patients must have direct bilirubin =\< 1.5 x institutional upper limit of normal (ULN) within 14 days prior to registration
* Patients must have alanine aminotransferase (ALT) =\< 3.0 x institutional upper limit of normal (ULN) or =\< 5.0 x ULN (if thought to be related to leukemic involvement) within 14 days prior to registration
* Prothrombin time (PT)/partial thromboplastin time (PTT)/ fibrinogen (as clinically indicated for example but not limited to history of bleeding or active bleeding, concern for disseminated intravascular coagulation) (within 14 days prior to registration to obtain baseline measurements)
* From metabolic panel (comprehensive or basic): sodium, potassium, chloride, carbon dioxide (CO2), and blood urea nitrogen (BUN) (within 14 days prior to registration to obtain baseline measurements)
* Patients must be able to safely discontinue use of strong inhibitors/inducers of CYP3A4 or PgP-g-p and must be able to safely discontinue use of naproxen for 48 hours before and after each dose of OBI-3424
* Patients with known human immunodeficiency virus (HIV)-infection are eligible providing they are on effective anti-retroviral therapy and have undetectable viral load at their most recent viral load test within 6 months prior to registration. (HIV viral load testing is required only for patients with known HIV infection). Patients must not be receiving antiviral therapies that are known strong inhibitors or inducers of CYP3A4
* Patients with evidence of chronic hepatitis B virus (HBV) infection may be eligible provided that they have an undetectable HBV viral load within 28 days prior to registration. Patients may be currently receiving HBV treatment. (HBV viral load testing is required only for patients with known HBV infection). Patients must not be receiving antiviral therapies that are known strong inhibitors or inducers of CYP3A4
* Patients with known history of hepatitis C virus (HCV) infection may be eligible provided that they have an undetectable HCV viral load within in 28 days prior to registration. Patients may be currently receiving treatment. (HCV viral load testing is required only for patients with known HCV infection). Patients must not be receiving antiviral therapies that are known strong inhibitors or inducers of CYP3A4
* Patients must not have a known history of prolonged QT interval by Fridericia (QTcF) (interval \> 450 msec for males; \> 470 msec for females). Patients that had transient prolongation of QTc secondary to medications or electrolyte abnormalities are not excluded if the QTc normalized and remain within acceptable QTcF range (interval \> 450 msec for males; \> 470 msec for females). Additionally, suspected medications should be no longer required or used, and electrolyte abnormalities must have normalized
* Patients must not be pregnant or nursing due to the teratogenic potential of the drug used on this study. Females of reproductive potential must have a negative serum pregnancy test within 14 days prior to registration. Women/men of reproductive potential must have agreed to use an effective contraceptive method during and up to 6 months after treatment. A woman is considered to be of "reproductive potential" if she has had menses at any time in the preceding 12 consecutive months. In addition to routine contraceptive methods, "effective contraception" also includes heterosexual celibacy and surgery intended to prevent pregnancy (or with a side-effect of pregnancy prevention) defined as a hysterectomy, bilateral oophorectomy or bilateral tubal ligation. However, if at any point a previously celibate patient chooses to become heterosexually active during the time period for use of contraceptive measures outlined in the protocol, he/she is responsible for beginning contraceptive measures
* Patients must not have other active malignancies for which they have received treatments within 6 months prior to registration excluding localized malignancies that do not require systemic treatment
* Patients must agree to have bone marrow and blood specimens submitted for MRD testing
* Patients must be offered the opportunity to participate in specimen banking. With patient consent, residuals from specimens submitted will be retained and banked for future research
* Patients must be informed of the investigational nature of this study and must sign and give written informed consent in accordance with fedral, local, institutional and Central Institutional Review Board (CIRB) guidelines unless they are unable to provide consent based on age (\< 18 years) or based on impaired decision-making capabilities. For patients \< 18 years of age or with impaired decision making capabilities, parents or other legally authorized representatives must sign and give informed consent on behalf of study participants in accordance with applicable federal, local, institutional and CIRB regulations
* As a part of the Oncology Patient Enrollment Network (OPEN) registration process the treating institution's identity is provided in order to ensure that the current (within 365 days) date of institutional review board approval for this study has been entered in the system
* This trial will use a slot reservation system to enroll the Phase I portion of the study. Patients planning to enroll at this phase of the study must first have a slot reserved in advance of the registration. All site staff will use OPEN to create a slot reservation DRUG: AKR1C3-activated Prodrug AST-3424, PROCEDURE: Biopsy Procedure, PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Marrow Aspiration, PROCEDURE: Computed Tomography
Recurrent T Acute Lymphoblastic Leukemia, Refractory T Acute Lymphoblastic Leukemia, Refractory T Lymphoblastic Lymphoma, T Lymphoblastic Lymphoma
Testing an Immunotherapy Anti-cancer Drug, Nivolumab, for Advanced Cancers in Patients With Autoimmune Disorders, AIM-NIVO (AIM-NIVO)
McFadden, Faith - mcfaddenfr@vcu.edu
Poklepovic, Andrew, S
NCT03816345
HM20022009
Inclusion Criteria:
* Patients can have either histologically confirmed malignancy that is radiologically evaluable and metastatic or unresectable, or have a malignancy for which a PD-1/PD-L1 inhibitor has been approved in the adjuvant setting, as well as the neoadjuvant or perioperative setting in which such treatment is considered standard of care or has been approved. Eligible tumor types include solid tumors and malignancies in which there is known evidence of clinical activity for single agent PD-1 or PD-L1 antibodies. Nivolumab or other PD1/PD-L1 inhibitors are FDA-approved for the treatment of melanoma, non-small cell lung cancer (NSCLC), Merkel cell cancer, bladder cancer, renal cell carcinoma (RCC), gastric cancer, hepatocellular carcinoma (HCC), cervical cancer, head and neck cancer, Hodgkin lymphoma (HL), metastatic small cell lung cancer (SCLC), and any solid tumor with microsatellite instability (MSI)-high status confirmed. Patients with HL are eligible but must follow standard response criteria. Additional tumor types may be eligible on a case by case basis upon discussion with principal investigator (PI)
* Patients enrolling on the trial for adjuvant use will be restricted to those with histology for which a PD-1/PD-L1 inhibitor has been approved in the adjuvant setting including but not limited to NSCLC, melanoma, RCC, cervical cancer, and bladder cancer
* Patients enrolled on the study can receive Nivolumab with other FDA-approved combinations according to the FDA package insert, including, but not limited to ipilimumab, cabozantinib or chemotherapy
* Patients who have previously received other forms of immunotherapy (high-dose \[HD\] IL-2, IFN, CTLA-4) are allowed. Patients must not have received cytokine immunotherapy for at least 4 weeks before nivolumab administration. Patients who have received prior anti-CTLA4 will be allowed and the washout period is 6 weeks
* Age \>= 18 years; children are excluded from this study but may be eligible for future pediatric phase 1 combination trials
* Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Karnofsky \>= 60)
* Life expectancy of greater than 12 weeks
* Leukocytes \>= 1,000/mcL
* Absolute neutrophil count \>= 500/mcL
* Platelets \>= 50,000/mcL
* Total bilirubin =\< 2 x institutional upper limit of normal (ULN)
* Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 5 x institutional ULN or =\< 8 x institutional ULN for patients with liver metastases or an autoimmune disease that is contributing to the elevation of these values
* Creatinine ULN OR glomerular filtration rate (GFR) \>= 30 mL/min (if using the Cockcroft-Gault formula)
* Human immunodeficiency virus (HIV)-infected patients on effective antiretroviral therapy with undetectable viral load within 6 months are eligible for this trial
* If evidence of chronic hepatitis B virus (HBV) infection, HBV viral load must be undetectable on suppressive therapy if indicated
* If history of hepatitis C virus (HCV) infection, must be treated with undetectable HCV viral load
* Patients with new or progressive brain metastases (active brain metastases) or leptomeningeal disease are eligible if the treating physician determines that immediate central nervous system (CNS) specific treatment is not required and is unlikely to be required for at least 4 weeks (or scheduled assessment after the first cycle of treatment), and a risk-benefit analysis (discussion) by the patient and the investigator favors participation in the clinical trial
* The effects of nivolumab on the developing human fetus are unknown. For this reason, women of child-bearing potential (WOCBP) and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. WOCBP receiving nivolumab will be instructed to adhere to contraception for a period of 5 months after the last dose of investigational product. Men receiving nivolumab and who are sexually active with WOCBP will be instructed to adhere to contraception for a period of 7 months after the last dose of investigational product
* Women of childbearing potential must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotropin \[HCG\]) within 24 hours prior to the start of nivolumab. Women must not be breastfeeding. Women who are not of childbearing potential (i.e., who are postmenopausal or surgically sterile as well as azoospermic men) do not require contraception
* WOCBP is defined as any female who has experienced menarche and who has not undergone surgical sterilization (hysterectomy or bilateral oophorectomy), tubal ligation, or who is not postmenopausal. Menopause is defined clinically as 12 months of amenorrhea in a woman over 45 in the absence of other biological or physiological causes. In addition, women under the age of 55 must have a documented serum follicle stimulating hormone (FSH) level less than 40 mIU/mL
* These durations have been calculated using the upper limit of the half-life for nivolumab (25 days) and are based on the protocol requirement that WOCBP use contraception for 5 half-lives plus 30 days, and men who are sexually active with WOCBP use contraception for 5 half-lives plus 90 days
* Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she (or the participating partner) should inform the treating physician immediately. Patients can resume treatment upon termination of a pregnancy or the completion of a successful pregnancy
* Ability to understand and the willingness to sign a written informed consent document
* Patients with more than one autoimmune disease are eligible. The treating physician would determine which autoimmune disease is dominant and the patient would be treated under that specific cohort (Please note: Patients with more than one autoimmune disease should receive assessments for all previously diagnosed autoimmune diseases. For example, a patient with psoriasis and IBD might be enrolled in the IBD cohort. Disease assessments for both psoriasis and IBD should be obtained, as per protocol. Case report forms \[CRFs\] for all relevant autoimmune diseases should be utilized. However, all additional cohort requirements will be considered optional and only the assessments from the assigned cohort will be considered mandatory)
* DM/SSc-SPECIFIC INCLUSION: Patients with known SSc or DM according to updated classification criteria (Van den Hoogan et al., Arthritis Rheum 2013;65(11):2737-47; Lundberg et al., A\&R in press). Overlap features are permitted, but patients must meet criteria for a "primary diagnosis" of DM or SSc
* DM/SSc-SPECIFIC INCLUSION: Patients may be on any concurrent therapy for DM or SSc unless specifically excluded
* DM/SSc-SPECIFIC INCLUSION: Patients must have a baseline computed tomography (CT) of the chest (within 6 months of study entry)
* RA-SPECIFIC INCLUSION: Rheumatologist-diagnosed RA requiring prior treatment with disease-modifying antirheumatic drugs (DMARDs) before patient was diagnosed with current malignancy. We recommend, but do not require, documentation for meeting 2010 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) classification criteria for RA
* RA-SPECIFIC INCLUSION: Prednisone up to 10 mg/day will be allowed. Intraarticular steroids will be allowed for the treatment of new symptomatic joints
* RA-SPECIFIC INCLUSION: Nonsteroidal anti-inflammatory drugs (NSAIDs) will be allowed
* SLE-SPECIFIC INCLUSION: SLE diagnosed by a rheumatologist. The patient should meet the revised 1997 American College of Rheumatology (ACR) classification criteria for SLE, but this is not mandatory
* ULCERATIVE COLITIS (UC)-SPECIFIC INCLUSION: Diagnosis of UC must be made by endoscopy with biopsies
* UC-SPECIFIC INCLUSION: Complete colonoscopy with biopsies during study screening, within 8 weeks before initial nivolumab administration, or within 4 weeks after initial nivolumab administration
* UC-SPECIFIC INCLUSION: Patients must test negative for hepatitis B (antigen \[Ag\] negative, antibody \[core (c)Ab\] negative, antibody \[surface (s)Ab\] positive or negative) and Mycobacterium tuberculosis (purified-protein- derivative \[PPD\] or enzyme-linked immunospot assay \[ELISpot or T-spot\]) or be on appropriate anti-microbial treatment for these infections
* UC-SPECIFIC INCLUSION: Mild Disease Cohort: Patients must be in clinical remission, defined as a Mayo Clinic score (MCS) of 2 or lower and no subscore higher than 1, and an endoscopic subscore of 0 or 1 either without medications, or treated with 5-ASA derivative, probiotic, or prior fecal transplant
* UC-SPECIFIC INCLUSION: Moderate Disease Cohort: Patients must be in clinical remission, defined as a MCS of 2 or lower and no subscore higher than 1, and an endoscopic subscore of 0 or 1 on 6-mercaptopurine, azathioprine, methotrexate, or rectal hydrocortisone, budesonide, or one of these medications in combination with any of the medications listed in the Mild cohort
* UC-SPECIFIC INCLUSION: Severe Disease Cohort (A or B): Patients must either be A) in clinical remission, defined as a MCS of 2 or lower and no subscore higher than 1, and an endoscopic subscore of 0 or 1 on a biologic therapy targeting tumor necrosis alpha (TNF-α) (infliximab, adalimumab, golimumab), α4β7 integrin (vedolizumab), or one of these biologic therapies in combination with any of the medications listed in the Mild or Moderate cohort, or B) have mild active disease defined as a MCS of 3-5 and no subscore higher than 2, and an endoscopic subscore of \< 2 on one of the medications or combination of medications defined for the Moderate or Mild cohort
* CROHN'S DISEASE (CD)-SPECIFIC INCLUSION: Complete colonoscopy with biopsies during study screening, within 8 weeks before initial nivolumab administration, or within 4 weeks after initial nivolumab administration
* CD-SPECIFIC INCLUSION: If patients have prior known disease in the stomach or small intestines, appropriate endoscopic evaluation (esophagogastroduodenoscopy/video capsule endoscopy) and/or imaging (computed tomography or magnetic resonance enterography) must also be current within 4 weeks prior to nivolumab administration
* CD-SPECIFIC INCLUSION: Deep enteroscopy techniques, such as double balloon enteroscopy, will not be required
* CD-SPECIFIC INCLUSION: Patients must test negative for hepatitis B (sAg negative, cAb negative, sAb positive or negative) and M. tuberculosis (PPD or ELISpot or T-spot) or be on appropriate anti-microbial treatment for these infections
* CD-SPECIFIC INCLUSION: Mild Disease Cohort: Patients must be in clinical remission as defined by a Crohn's Disease Activity Index (CDAI) \< 150 either without treatment or on a 5-ASA derivative, probiotic, antibiotics, or following fecal transplant
* CD-SPECIFIC INCLUSION: Moderate Disease Cohort: Patients must be in clinical remission as defined by a CDAI \< 150 on 6-mercaptopurine, azathioprine, methotrexate, rectal hydrocortisone, budesonide, or one of these medications in combination with any of the medications listed in the Mild cohort
* CD-SPECIFIC INCLUSION: Severe Disease Cohort (A or B): Patients must either A) be in clinical remission as defined by a CDAI \< 150 on biologic therapy targeting TNF-α (infliximab, adalimumab, certolizumab pegol), IL-12/23p40 (ustekinumab), α4β7 integrin (vedolizumab), or one of these biologic therapies in combination with any of the medications listed in the Mild or Moderate cohort, or B) have mild active disease as defined by a CDAI of 150 to 220 on one of medications or combination of medications defined for the Moderate or Mild cohort
* OTHER AUTOIMMUNE DISEASES- NS-SPECIFIC INCLUSION: For other autoimmune diseases that cannot be classified, the eligibility criteria will be determined by the managing rheumatologist or other autoimmune disease specialist, based on the clinical judgement and current American College of Radiology (ACR) classification guidelines or other relevant guidelines, as per the disease category in question
* OTHER AUTOIMMUNE DISEASES- NS-SPECIFIC INCLUSION: For giant cell arteritis (GCA), patients must have had positive temporal artery biopsy for GCA and abnormal erythrocyte sedimentation rate (ESR) at time of diagnosis
* OTHER AUTOIMMUNE DISEASES- NS-SPECIFIC INCLUSION: For polymyalgia rheumatica (PMR), patients must have clinical diagnosis in addition to elevated inflammatory markers including (ESR, C reactive protein \[CRP\])
* OTHER AUTOIMMUNE DISEASES- NS-SPECIFIC INCLUSION: Patients can be in remission (with no glucocorticoids or immunosuppressive medications) or have low-moderate activity, which is defined as being on prednisone ≤ 10 mg or equivalent
* MS-SPECIFIC INCLUSION: Patients must meet 2017 McDonald criteria for the diagnosis of MS (Thompson AJ, et al. Diagnosis of multiple sclerosis: 2017 revision of the McDonald criteria. Lancet Neurol. 17(2):162-173.)
* MS-SPECIFIC INCLUSION: Patients with MS can be in remission and can have a history of being on immunomodulatory agents, but at the time of entry into the clinical trial, patients should be off any concurrent MS therapy for at least 2 weeks. Patients receiving concomitant interferon gamma (IFN-γ treatment) will be permitted in the study
* SJS-SPECIFIC INCLUSION: SjS diagnosed by a rheumatologist or oral medicine provider. The patient should meet the American-European Consensus Criteria for Sjögren's Syndrome (Vitali, et al., 2002). If on treatment, the patient may only be on hydroxychloroquine and prednisone ≤ 10 mg or equivalent
* PSO/PSA-SPECIFIC INCLUSION: Patients with known PsO as diagnosed by a dermatologist or PsA by a rheumatologist and/or by Classification for Psoriatic Arthritis (CASPAR) criteria (Tillett et al., 2012)
* PSO/PSA-SPECIFIC INCLUSION: Patients must have stable disease as determined by the investigator with no change in systemic therapy and/or biologic therapy for at least 3 months, except for those on tumor necrosis factor (TNF) inhibitors. In the case of TNF inhibition, patients may have transitioned to an alternative biologic therapy with stable disease for at least 4 weeks. For PsA, no change in corticosteroid therapy for at least 1 month prior to baseline and dose must be 10 mg or less
* PSO/PSA-SPECIFIC INCLUSION: Patients may be on any concurrent therapy for PsO or PsA unless specifically excluded
Exclusion Criteria:
* Patients who have had chemotherapy or radiotherapy within 2 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events (AEs) due to agents administered more than 4 weeks earlier have not resolved or stabilized. Palliative (limited-field) radiation therapy (RT) is permitted (2 week washout from start of treatment), if all of the following criteria are met:
* Repeat imaging demonstrates no new sites of bone metastases
* The lesion being considered for palliative radiation is not a target lesion
* Patients with prior therapy with an anti-PD-1 or anti-PD-L1
* Patients with prior allogeneic hematologic transplant
* Patients who are receiving any other anticancer investigational agents
* Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
* UC-SPECIFIC EXCLUSION: Patients who have received ipilimumab treatment
* UC-SPECIFIC EXCLUSION: Prior colectomy
* UC-SPECIFIC EXCLUSION: Concurrent primary sclerosing cholangitis (PSC). Patients with PSC can be enrolled on the Other Autoimmune Diseases Cohorts
* UC-SPECIFIC EXCLUSION: Patients on empiric immunosuppressive treatment without any clinical workup
* CD-SPECIFIC EXCLUSION: Known untreated abscesses, untreated and symptomatic strictures, short gut physiology, or isolated jejunal disease
* CD-SPECIFIC EXCLUSION: Patients who have received ipilimumab treatment
* CD-SPECIFIC EXCLUSION: Patients on empiric immunosuppressive treatment without any clinical workup
* MS-SPECIFIC EXCLUSION: Patients with MS cannot have medical contraindications to gadolinium-enhanced magnetic resonance imaging (MRI) PROCEDURE: Biospecimen Collection, DRUG: Cabozantinib, DRUG: Fluoropyrimidine, BIOLOGICAL: Ipilimumab, BIOLOGICAL: Nivolumab, DRUG: Platinum Compound, DRUG: Platinum Doublet
Autoimmune Disease, Crohn Disease, Dermatomyositis, Hematopoietic and Lymphoid Cell Neoplasm, Inflammatory Bowel Disease, Malignant Solid Neoplasm, Multiple Sclerosis, Psoriasis, Psoriatic Arthritis, Rheumatoid Arthritis, Sjogren Syndrome, Systemic Lupus Erythematosus, Systemic Scleroderma, Ulcerative Colitis
Testing What Happens When an Immunotherapy Drug (Pembrolizumab) is Given by Itself Compared to the Usual Treatment of Chemotherapy With Radiation After Surgery for Recurrent Head and Neck Squamous Cell Carcinoma
Schaefer, Melissa - schaeferm@vcu.edu
Alesi, Erin, R.
NCT04671667
HM20022077
Inclusion Criteria:
* Patient must be between 18 and 79 years of age
* Patient must have locoregionally recurrent or second primary HNSCC (oral cavity, oropharynx, larynx, hypopharynx) in a previously radiated field
* Patient must have undergone surgery with gross total resection and must be randomized within 8 weeks of surgery
* Patients must have high risk disease defined as:
* Positive margins and/or extra nodal extension (ENE)
* Positive margins are defined as malignancy at or within 1 mm of the margin. High grade dysplasia (i.e. carcinoma in situ) at the margin is also considered positive
* ENE may be either gross or microscopic
* Patient must have a PD-L1 Combined Positive Score (CPS) \>= 1 in a Clinical Laboratory Improvement Act (CLIA) certified laboratory. Testing can be done locally as long as it is done in a CLIA certified laboratory. This testing must be on the tumor specimen from the resection of the patient's recurrent or second primary HNSCC
* Patient must have had prior radiation to the area of recurrent or second primary tumor. This is defined as \> 50% of the presurgical tumor volume having previously received a dose of \> 45 Gy as determined by the treating radiation oncologist
* Patient must have completed prior radiation a minimum of 6 months prior to randomization
* Patient must not have any evidence of distant disease based on baseline imaging done within 28 days prior to randomization
* Patient must not have received anti-PD-1/PD-L1 therapy for recurrent disease. If the patient received anti-PD-1/PD-L1 therapy as part of initial upfront curative intent treatment (either as part of definitive non-surgical therapy or in the adjuvant setting) in the past, the last dosage of anti-PD-1/PD-L1 therapy must have been given greater than one year prior to randomization
* Patient must have an Eastern Cooperative Oncology Group (ECOG) performance status 0-1
* Patient must have the ability to understand and the willingness to sign a written informed consent document. Patients with impaired decision-making capacity (IDMC) who have a legally authorized representative (LAR) or caregiver and/or family member available will also be considered eligible
* Patient must not be pregnant or breast-feeding due to the potential harm to an unborn fetus and possible risk for adverse events in nursing infants with the treatment regimens being used. All patients of childbearing potential must have a blood test or urine study within 14 days prior to randomization to rule out pregnancy. A urine or serum pregnancy test must be repeated within 72 hours prior to receiving the first dose of pembrolizumab or chemotherapy if the test done for eligibility/randomization is done outside of this 72 hour window. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. A patient of childbearing potential is someone, regardless of whether they have undergone tubal ligation, who meets the following criteria: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy; or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months)
* Patient must not expect to conceive or father children by using by using accepted and effective method(s) of contraception or by abstaining from sexual intercourse while on study treatment, and continue for 120 days after the last dose of study treatment
* Absolute neutrophil count (ANC) \>= 1,500/mcL (obtained =\< 28 days prior to protocol randomization)
* Platelets \>= 100,000/mcL (obtained =\< 28 days prior to protocol randomization)
* Total bilirubin =\< 1.5 x institutional upper limit of normal (ULN) (obtained =\< 28 days prior to protocol randomization)
* Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) =\< 3.0 x institutional ULN (obtained =\< 28 days prior to protocol randomization)
* Creatinine clearance \> 30 ml/min using the Cockcroft-Gault formula (obtained =\< 28 days prior to protocol randomization)
* Patient must not have a current active infection that requires systemic treatment at time of randomization
* Patient must not have a history of non-infectious pneumonitis requiring steroids within 3 years prior to randomization
* Patient must not have a history of solid organ transplant or stem cell transplant
* Patient must not be on immunosuppressive medication within 7 days prior to randomization, EXCEPT for the following: a) intranasal, inhaled, topical steroids, or local steroid injection (e.g., intra-articular injection); b) systemic corticosteroids at physiologic doses =\< 10 mg/day of prednisone or equivalent; c) steroids as premedication for hypersensitivity reactions (e.g., CT scan premedication)
* Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional classification. Patients with New York Heart Association class III or IV heart failure are not eligible
* Patient must not have received a live vaccine within 30 days prior to the first dose of study drug. Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guerin (BCG), and typhoid vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (e.g., FluMist \[registered trademark\]) are live attenuated vaccines and are not allowed
* Patient must not have severe hypersensitivity (\>= grade 3) to pembrolizumab and/or any of its excipients
* Patient must not have an active autoimmune disease that has required systemic treatment in past 2 years (i.e., with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency) is not considered a form of systemic treatment and is allowed
* Patient must not have a known psychiatric or substance abuse disorder that would interfere with the participant's ability to cooperate with the requirements of the study
* Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial as long as they have not been HIV-infected with a history of Kaposi sarcoma and/or multicentric Castleman disease
* Patient must not have a known history of hepatitis B (defined as hepatitis B surface antigen \[HBsAg\] reactive) or known active hepatitis C virus (defined as HCV ribonucleic acid \[RNA\] \[qualitative\] is detected) infection
* NOTE: No testing for hepatitis B and hepatitis C is required unless mandated by a local health authority DRUG: Carboplatin, DRUG: Cisplatin, PROCEDURE: Computed Tomography, RADIATION: Intensity-Modulated Radiation Therapy, PROCEDURE: Magnetic Resonance Imaging, BIOLOGICAL: Pembrolizumab, RADIATION: Proton Beam Radiation Therapy
Recurrent Head and Neck Squamous Cell Carcinoma, Recurrent Hypopharyngeal Squamous Cell Carcinoma, Recurrent Laryngeal Squamous Cell Carcinoma, Recurrent Oral Cavity Squamous Cell Carcinoma, Recurrent Oropharyngeal Squamous Cell Carcinoma
Examining the Impact of Exercise Training on Vascular Dysfunction in Individuals With Mental Health Disorders - Study 3
Ryan Garten, PhD - rsgarten@vcu.edu
NCT04916340
Inclusion Criteria:
* apparently healthy and free of overt cardiovascular, pulmonary, or metabolic disease
* for PTSD group, a score of ≥ 33 on PCL-5 checklist
* for GAD group, a score of ≥ 10 on the GAD-7 self-report scale and \< 33 on the PCL-5 checklist
* for Healthy Control group, a score of ≤ 10 on the GAD-7 self-report scale and \< 33 on the PCL-5 checklist
Exclusion Criteria:
* taking medications that could influence cardiovascular function
* current smokers who have recently quit smoking
* illicit drug use or excessive alcohol consumption
* pregnant women
* significant calorie restriction or vitamin/mineral deficiencies
* limited English proficiency BEHAVIORAL: Muscular Strength Training Group, BEHAVIORAL: Muscular Fitness Training Group
Peripheral Vascular Diseases
cardiovascular disease, vascular function, PTSD, GAD, Oxidant
Examining the Impact of Exercise Training on Vascular Dysfunction in Individuals With Mental Health Disorders - Study 1
Ryan Garten, PhD - rsgarten@vcu.edu
NCT04916327
Inclusion Criteria:
* apparently healthy and free of overt cardiovascular, pulmonary, or metabolic disease
* for PTSD group, a score of ≥ 33 on PCL-5 checklist
* for GAD group, a score of ≥ 10 on the GAD-7 self-report scale and \< 33 on the PCL-5 checklist
* for Healthy Control group, a score of ≤ 10 on the GAD-7 self-report scale and \< 33 on the PCL-5 checklist
Exclusion Criteria:
* taking medications that could influence cardiovascular function
* current smokers who have recently quit smoking
* illicit drug use or excessive alcohol consumption
* pregnant women
* significant calorie restriction or vitamin/mineral deficiencies
* limited English proficiency DIETARY_SUPPLEMENT: Antioxidant, DIETARY_SUPPLEMENT: Placebo
Peripheral Vascular Diseases
cardiovascular disease, vascular function, PTSD, GAD, Oxidant
Examining the Impact of Exercise Training on Vascular Dysfunction in Individuals With Mental Health Disorders - Study 2
Ryan Garten, PhD - rsgarten@vcu.edu
NCT04922762
Inclusion Criteria:
* apparently healthy and free of overt cardiovascular, pulmonary, or metabolic disease
* for PTSD group, a score of ≥ 33 on PCL-5 checklist
* for GAD group, a score of ≥ 10 on the GAD-7 self-report scale and \< 33 on the PCL-5 checklist
* for Healthy Control group, a score of ≤ 10 on the GAD-7 self-report scale and \< 33 on the PCL-5 checklist
Exclusion Criteria:
* taking medications that could influence cardiovascular function
* current smokers who have recently quit smoking
* illicit drug use or excessive alcohol consumption
* pregnant women
* significant calorie restriction or vitamin/mineral deficiencies
* limited English proficiency BEHAVIORAL: Moderate Intensity, Normal Volume Exercise Training, BEHAVIORAL: High Intensity, Normal Volume Exercise Training, BEHAVIORAL: Moderate Intensity, High Volume Exercise Training
Peripheral Vascular Diseases
cardiovascular disease, vascular function, PTSD, GAD, Oxidant
A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 Diabetes
Medical Information - medicalinfo@vrtx.com
Levy, Marlon
NCT04786262
HM20021228
Key
Inclusion Criteria:
* Clinical history of T1D with \> 5 years of duration of insulin dependence
* At least two episodes of documented severe hypoglycemia in the 12 months prior to enrollment
* Stable diabetic treatment
* Consistent use of continuous glucose monitor (CGM) for at least 3 months before Screening and willingness to use CGM for the duration of the study
Key Exclusion Criteria:
-Prior islet cell transplant, organ transplant, or cell therapy
Other protocol defined Inclusion/Exclusion criteria may apply BIOLOGICAL: VX-880
Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia
Testing the Use of Combination Therapy in Adult Patients With Newly Diagnosed Multiple Myeloma, the EQUATE Trial (EQUATE)
Lantis, Kristin - kllantis@vcu.edu
Yazbeck, Victor, Y
NCT04566328
HM20022350
Inclusion Criteria:
* STEP 0 - Patient must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0-2 (PS 3 allowed if secondary to pain)
* STEP 0 - Patient must have newly diagnosed multiple myeloma (MM) by International Myeloma Working Group (IMWG) criteria
* STEP 0 - Patient must agree to register to the mandatory REVLIMID Risk Evaluation and Mitigation Strategy (RevREMS) program and be willing and able to comply with the requirements of RevREMS
* STEP 0 - Patient must be able to undergo diagnostic bone marrow aspirate following preregistration.
* NOTE: Bone marrow aspirate specimen must be submitted to Adaptive Biotechnologies for clonoSEQ Assay
* NOTE: Adaptive Biotechnologies will release results to the diagnostic Portal from the Clonality (ID) test within fourteen (14) days of receipt and reconciliation of fresh bone marrow specimen to the submitting institution
* STEP 1 - Patient must meet all eligibility criteria in STEP 0 with exception of allergy requirement
* STEP 1 - Institution must have received the Clonality (ID) test results from Adaptive Biotechnologies and dominant sequences were identified
* STEP 1 - Patient must have standard risk MM as defined by the Revised International Staging System (RISS) stage I or II
* NOTE: R-ISS stage is based on serum beta2 microglobulin, albumin and lactate dehydrogenase (LDH) levels along with presence of chromosomal abnormalities (CA) detected by interphase fluorescent in situ hybridization (iFISH). Presence of del(17p), t(4;14), and/or t(14;16) is considered high risk and absence of these, including any other findings, are standard risk
* R-ISS stage
* Stage I: ISS stage I \[beta2 macroglobulin \< 3.5 mg/L, albumin \> 3.5 g/dL\] AND standard-risk CA AND normal LDH
* Stage II: Not R-ISS stage I or III
* Stage III: ISS stage III \[beta2 macroglobulin \> 5.5 mg/L\] AND high-risk CA OR high LDH (\> upper limit of normal) \[patients with stage III are ineligible\]
* STEP 1 - Patient must have measurable or evaluable disease as defined by having one or more of the following, obtained within 28 days prior to registration:
* \>= 1 g/dL monoclonal protein (M-protein) on serum protein electrophoresis
* \>= 200 mg/24 hours of monoclonal protein on a 24-hour urine protein electrophoresis
* Involved free light chain \>= 10 mg/dL or \>= 100 mg/L AND abnormal serum immunoglobulin kappa to lambda free light chain ratio (\< 0.26 or \> 1.65)
* Monoclonal bone marrow plasmacytosis \>= 30% (evaluable disease)
* STEP 1 - Patients must have a serum protein electrophoresis (SPEP), urine protein electrophoresis (UPEP), and serum free light chain (FLC) assay performed within 28 days prior to registration. In addition, a bone marrow biopsy and/or aspirate is required within 28 days if bone marrow is being followed for response
* NOTE: UPEP (on a 24-hour collection) is required, no substitute method is acceptable. Urine must be followed monthly if the baseline urine M-spike is \>= 200 mg/24 hr. Please note that if both serum and urine M-components are present, both must be followed in order to evaluate response
* NOTE: The serum free light chain test is required to be done if the patient does not have measurable disease in the serum or urine. Measurable disease in the serum is defined as having a serum M-spike \>= 1 g/dL. Measurable disease in the urine is defined as having a urine M-spike \>= 200 mg/24 hr
* STEP 1 - Calculated creatinine clearance \> 30 mL/min (obtained =\< 14 days prior to Step 1 registration)
* STEP 1 - Absolute neutrophil count (ANC) \>= 1000/mm\^3 (obtained =\< 14 days prior to Step 1 registration)
* STEP 1 - Untransfused platelet count \>= 75,000/mm\^3 (obtained =\< 14 days prior to Step 1 registration)
* STEP 1 - Hemoglobin \>= 8.0 g/dL (obtained =\< 14 days prior to Step 1 registration)
* STEP 1 - Total bilirubin =\< 1.5 x ULN (institutional upper limit of normal) (obtained =\< 14 days prior to Step 1 registration)
* STEP 1 - Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =\< 3 x ULN (obtained =\< 14 days prior to Step 1 registration)
* STEP 1 - Patient must have received no more than one cycle (28 days or less) of prior chemotherapy and no more than 160 mg of prior dexamethasone (or equivalent dose of prednisone) for treatment of symptomatic myeloma. Patient must not have been exposed to daratumumab for treatment of symptomatic myeloma. Prior radiation therapy to symptomatic lesions is allowed provided there are no residual toxicity related to radiation and blood counts meet the study requirements. Radiation treatment must be completed at least 14 days prior to Step 1 registration
* STEP 1 - Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months of randomization are eligible for this trial
* STEP 1 - For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
* STEP 1 - Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
* STEP 1 - Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
* STEP 1 - Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class 2B or better. Patients must not have evidence of current uncontrolled cardiovascular conditions, including hypertension, cardiac arrhythmias, congestive heart failure, unstable angina, or myocardial infarction within 6 months prior to Step 1 registration
* STEP 1 - Patient may have a history of current or previous deep vein thrombosis (DVT) or pulmonary embolism (PE) but must be willing to take some form of anti-coagulation as prophylaxis if they are not currently on full-dose anticoagulation
* STEP 1 - Patients with a history of chronic obstructive pulmonary disease (COPD) must have FEV1 testing done within 28 days prior to Step 1 registration and the forced expiratory volume in 1 second (FEV1) must be \> 50% of predicted normal
* STEP 2 - Institution must have received Tracking (MRD) test results from Adaptive Biotechnologies
* STEP 2 - Patient must have completed the Step 1 Induction phase of this protocol without experiencing progression
* STEP 2 - Patient must be registered to Step 2 within 8 weeks of completing Step 1 Induction Treatment, counting from last day of completion of last cycle
* STEP 2 - Patient must have an ECOG performance status (PS) of 0-2 (PS 3 allowed if secondary to pain)
* STEP 2 - Any adverse event(s) related to Step 1 Induction Treatment must have resolved to grade 2 or less
* STEP 2 - Hemoglobin \>= 8 g/dL (obtained within 14 days prior to Step 2 randomization)
* STEP 2 - Platelet count \>= 50,000/mm\^3 (obtained within 14 days prior to Step 2 randomization)
* STEP 2 - Absolute neutrophil count (ANC) \>= 1000/mm\^3 (obtained within 14 days prior to Step 2 randomization)
* STEP 2 - Calculated creatinine clearance \>= 30 mL/min (obtained within 14 days prior to Step 2 randomization)
* STEP 2 - Total bilirubin =\< 1.5 x ULN (Institutional upper limit of normal) (obtained within 14 days prior to Step 2 randomization)
* STEP 2 - ALT and AST \< 3 x ULN (obtained within 14 days prior to Step 2 randomization)
Exclusion Criteria:
* STEP 0 - Patient must not have any known allergies, hypersensitivity, or intolerance to corticosteroids, monoclonal antibodies or human proteins, or their excipients (refer to respective package inserts or Investigator's Brochure), or known sensitivity to mammalian-derived products
* STEP 1 - Women must not be pregnant or breast-feeding due to the potential harm and teratogenic effects to an unborn fetus and possible risk for adverse events in nursing infants with the treatment regimens being used. All females of childbearing potential must have a blood test or urine study with a sensitivity of at least 25 mIU/mL within 10-14 days prior to Step 1 registration to rule out pregnancy and again within 24 hours prior to the first dose of lenalidomide. Females of childbearing potential must also agree to ongoing pregnancy testing while on protocol treatment. A female of childbearing potential is defined as any woman, regardless of sexual orientation or whether they have undergone tubal ligation, who meets the following criteria:
* Has achieved menarche at some point,
* Has not undergone a hysterectomy or bilateral oophorectomy; or
* Has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months)
* STEP 1 - Women of childbearing potential must not expect to conceive children by using accepted and effective method(s) of contraception (for this protocol defined as the use of TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME for 1) at least 28 days before starting protocol treatment; 2) while participating in the study; 3) during dose interruptions; and 4) for at least 3 months days after the last dose of protocol treatment) OR by practicing true abstinence from sexual intercourse for the duration of their participation in the study (periodic abstinence \[e.g., calendar, ovulation, symptothermal, post-ovulation methods\] and withdrawal are not acceptable methods of contraception). Men must not expect to father children by practicing true abstinence from sexual intercourse for the duration of their participation in the study (periodic abstinence \[e.g., calendar, ovulation, symptothermal, post-ovulation methods\] and withdrawal are not acceptable methods of contraception) OR use a latex condom during sexual contact with a female of child bearing potential while participating in the study and for at least 3 months after the last dose of protocol treatment even if they have had a successful vasectomy. Men must also agree to abstain from donating sperm while on study treatment and for 3 months after the last dose of protocol treatment even if they have had a successful vasectomy. Both women and men must both agree to abstain from donating blood during study participation and for at least 28 days after the last dose of protocol treatment
* STEP 1 - Patient must not have peripheral neuropathy \>= grade 2 on clinical examination or grade 1 with pain at time of Step 1 registration
* STEP 1 - Patient must not have any serious medical or psychiatric illness that could, in the investigator's opinion, potentially interfere with the completion of treatment according to this protocol
* STEP 1 - Patient must not have moderate or severe persistent asthma within the past 2 years, or uncontrolled asthma of any classification
* NOTE: Patients who currently have controlled intermittent asthma or controlled mild persistent asthma are allowed to register
* STEP 1 - Patient must not receive any other concurrent chemotherapy, or any ancillary therapy considered investigational while on this protocol
* NOTE: Bisphosphonates are considered to be supportive care rather than therapy, and are thus allowed while on protocol treatment
* STEP 2 - Patient must not have received any non-protocol therapy outside of the assigned Step 1 Induction treatment including stem cell transplant
* STEP 2 - Women must not be pregnant or breast-feeding due to the potential harm and teratogenic effects to an unborn fetus and possible risk for adverse events in nursing infants with the treatment regimens being used. All females of childbearing potential must have a blood test or urine study with a sensitivity of at least 25 mIU/mL within 10-14 days prior to Step 2 randomization to rule out pregnancy and again within 24 hours prior to the first dose of lenalidomide. Females of childbearing potential must also agree to ongoing pregnancy testing while on protocol treatment. A female of childbearing potential is defined as any woman, regardless of sexual orientation or whether they have undergone tubal ligation, who meets the following criteria:
* Has achieved menarche at some point,
* Has not undergone a hysterectomy or bilateral oophorectomy; or
* Has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).
* STEP 2 - Women of childbearing potential must not expect to conceive children by using accepted and effective method(s) of contraception (for this protocol defined as the use of TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME for 1) at least 28 days before starting protocol treatment; 2) while participating in the study; 3) during dose interruptions; and 4) for at least 3 months days after the last dose of protocol treatment) OR by practicing true abstinence from sexual intercourse for the duration of their participation in the study (periodic abstinence \[e.g., calendar, ovulation, symptothermal, post-ovulation methods\] and withdrawal are not acceptable methods of contraception).
Men must not expect to father children by practicing true abstinence from sexual intercourse for the duration of their participation in the study (periodic abstinence \[e.g., calendar, ovulation, symptothermal, post-ovulation methods\] and withdrawal are not acceptable methods of contraception) OR use a latex condom during sexual contact with a female of child bearing potential while participating in the study and for at least 3 months after the last dose of protocol treatment even if they have had a successful vasectomy. Men must also agree to abstain from donating sperm while on study treatment and for 3 months after the last dose of protocol treatment even if they have had a successful vasectomy. Both women and men must both agree to abstain from donating blood during study participation and for at least 28 days after the last dose of protocol treatment DRUG: Bortezomib, BIOLOGICAL: Daratumumab and Hyaluronidase-fihj, DRUG: Dexamethasone, DRUG: Lenalidomide, OTHER: Quality-of-Life Assessment
Plasma Cell Myeloma, RISS Stage I Plasma Cell Myeloma, RISS Stage II Plasma Cell Myeloma
Effects of Dexrazoxane Hydrochloride on Biomarkers Associated With Cardiomyopathy and Heart Failure After Cancer Treatment
Gwaltney, Lindsey - lbgwaltney@vcu.edu
Gowda, Madhu, S
NCT01790152
HM20000463
Inclusion Criteria:
Study Strata I, II, and III are closed for further patient entry as of March 31, 2021. The study remains open for existing medical record submission of Stratum IV
* STRATUM I AND STRATUM II: LEUKEMIA AND LYMPHOMA SURVIVORS
* Previously enrolled leukemia and lymphoma survivors, randomized to + or - DRZ on P9404, P9425, P9426, or DFCI 95-01 (high-risk patients only)
* STRATUM I: Alive and in continuous first complete remission from their original cancer (leukemia/lymphoblastic lymphoma \[P9404, high-risk DFCI 95-01\] or Hodgkin lymphoma \[P9425/P9426\])
* STRATUM I: Did not have progressive disease or induction failure requiring off-protocol therapy including hematopoietic cell transplantation
* STRATUM I: Must not have been diagnosed with any subsequent malignancy that required additional cardiotoxic therapies (i.e., radiotherapy to the chest \[also includes fields directed towards the neck, upper abdomen, or spine\], or additional anthracyclines or anthraquinones); patients with history of subsequent malignancy that did not require such therapies remain eligible
* STRATUM I: All patients and/or their parents or legal guardians must sign a written informed consent
* STRATUM II: Among leukemia and lymphoma patients randomized to + or - DRZ on P9404, P9425, P9426, and DFCI 95-01 (high risk patients only) who have relapsed or have experienced a subsequent malignancy that precludes eligibility since their original diagnosis, the study committee will review the available data (both from Children's Oncology Group's \[COG?s\] Statistics and Data Center \[SDC\] and the participating institution) to determine if individual patients are to be selected for Stratum 2; in recognition that local institutions sometimes have more updated relapse/subsequent cancer data than SDC, in cases where local data is more updated, local data will be used preferentially; the study will petition the Institutional Review Board (IRB) specifically for a waiver of consent to include any relapse and subsequent cancer data obtained from existing records for analysis of the secondary aims; patients selected for Stratum 2 will be those for whom late relapse or subsequent cancer is reported but who lack clear confirmation in existing records (either at SDC or at the local institution)
* STRATUM II: Alive, but have experienced relapse of their original cancer and/or have developed a subsequent cancer (other than non-melanomatous skin cancer) since their original diagnosis
* STRATUM II: All patients and/or their parents or legal guardians must sign a written informed consent
* STRATUM III: OSTEOSARCOMA SURVIVORS
* Previously enrolled osteosarcoma survivors treated on P9754 who are alive and able (themselves and/or parents/legal guardian) to provide written informed consent; note that relapse and subsequent malignancy are not exclusion criteria for P9754 survivors
* Comparison subjects for P9754 survivors will be eligible to be enrolled from any ALTE11C2 participating COG site (even if that institution did not participate on P9754), according to the following criteria:
* Newly diagnosed, previously untreated biopsy-proven moderate or high grade osteosarcoma without metastasis; patients with low grade osteosarcoma, parosteal or periosteal sarcoma are ineligible
* \< 31 years of age at time of initial osteosarcoma diagnosis
* Diagnosis occurred between January 1, 1999 through December 31, 2002; duration of therapy can extend beyond 2002
* No evidence of poor or low cardiac function at time of initial osteosarcoma diagnosis; if reports from the time are available: shortening fraction \>= 28% by echocardiogram and within the institutional normative range for age, or radionuclide angiogram ejection fraction \>= 50%; if imaging reports from the time are no longer available, there must be no documentation within available medical records that suggest poor or low cardiac function at time of diagnosis
* Comparison subject must have institutional records (e.g., clinic note, treatment summary, chemotherapy roadmap) documenting lifetime receipt of 450 to 600 mg/m\^2 of doxorubicin (doses within 10% are acceptable); this includes initial therapy as well as any subsequent therapy for relapse or second cancer, if relevant; as such, comparison subjects who have had osteosarcoma relapse or subsequent malignancies remain eligible so long as they meet all other eligibility criteria
* No anthracycline or anthraquinone aside from doxorubicin was ever given as part of initial or subsequent therapies
* No exposure to DRZ at any point in time
* All patients and/or their parents or legal guardians must sign a written informed consent
* STRATUM IV: CARDIOMYOPATHY CASES, NOT OTHERWISE ELIGIBLE FOR STRATUMS 1, 2, AND 3
* Individuals diagnosed with cancer prior to age 21 years, who required treatment with chemotherapy and/or radiotherapy, achieved initial remission, and remained alive after completing anti-cancer-therapy for at least 1 year
* Must have screening echocardiograms for heart function as part of cancer therapy and off-therapy evaluations available (Digital Imaging and Communications in Medicine \[DICOM\] format). Images from Video Home System (VHS) tapes and reports only (without images) are not suitable
* Cannot have a known history of congenital heart disease (patent foramen ovale remain eligible) or underlying genetic syndrome associated with abnormal cardiovascular development or health (e.g., down syndrome)
* Based on echocardiography, must have either left ventricular fractional shortening =\< 28.0% or ejection fraction =\< 50.0% on at least two occasions, with at least one of these measurements occurring after cancer therapy completion and be in the absence of sepsis or any uncontrolled infection
* If the fractional shortening or ejection fraction criteria is only met on one occasion, this must be after cancer therapy completion, be in the absence of sepsis or any uncontrolled infection, and the patient must have subsequently started on chronic medical therapy for cardiomyopathy (e.g., beta-blocker, angiotensin-converting enzyme \[ACE\]-inhibitor, angiotensin receptor blocker) lasting at least 6 months
* For all participants (stratums 1, 2, 3, and 4), all institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met OTHER: Assessment of Therapy Complications, OTHER: Laboratory Biomarker Analysis, OTHER: Quality-of-Life Assessment, OTHER: Questionnaire Administration
Hodgkin Lymphoma in Remission, Leukemia in Remission, Lymphoblastic Lymphoma, Osteosarcoma, Recurrent Leukemia, Recurrent Lymphoma, Recurrent Malignant Neoplasm
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X)
Howell, Jodie - jodie.howell@vcuhealth.org
Johnson, Nicholas, E
NCT05004129
HM20023901
Inclusion Criteria:
Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:
• Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
• Diagnosis must be genetically confirmed
• Subjects must be male or female aged ≥6 years to ≤45 years at Screening
• Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
• Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations)
• Subject's caregiver must be willing and able to support participation for duration of study
• Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol Subjects entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered eligible for the study without meeting all of the criteria below:
• Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
• Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or LAR provides consent, there must also be assent from the subject (as required by local regulations)
• Subject's caregiver must be willing and able to support participation for duration of study
• Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol Key
Exclusion Criteria:
• Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
• New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit
• Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
• Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
• Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months other than the AMO-02- MD-2-003 study
• Existing or historical medical conditions or complications (eg. neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine or respiratory disease) that may impact the interpretability of the study results
• Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry
DRUG: Tideglusib
Congenital Myotonic Dystrophy
Tideglusib, AMO-02-MD-2-004, Congenital Myotonic Dystrophy, Myotonic Dystrophy, Dystrophia Myotonica, Myotonia Atrophica, Myotonia Dystrophica, Myotonic Dystrophy, Congenital, Steinert Disease, Steinert Myotonic Dystrophy, Steinert's Disease
Trifecta-Kidney cfDNA-MMDx Study
Konrad S Famulski, PhD - konrad@ualberta.ca
NCT04239703
Inclusion Criteria:
* All kidney transplant recipients undergoing a kidney biopsy for clinical indications, as determined by their physician or surgeon, will be eligible to enroll in the study.
Exclusion Criteria:
* Patients will be excluded from the study if they decline participation or are unable to give informed consent or multiple organ recipients. DIAGNOSTIC_TEST: MMDx, DIAGNOSTIC_TEST: Prospera, DIAGNOSTIC_TEST: HLA antibody
Kidney Transplant Rejection
donor derived cell-free DNA, blood, kidney biopsy
Testing the Addition of the Drug Apalutamide to the Usual Hormone Therapy and Radiation Therapy After Surgery for Prostate Cancer, INNOVATE Trial (INNOVATE)
Loney, Shenise - loneys2@vcu.edu
Urdaneta, Alfredo, I
NCT04134260
HM20021914
Inclusion Criteria:
* Pathologically (histologically) proven diagnosis of prostate adenocarcinoma. Any type of radical prostatectomy is permitted, including retropubic, perineal, laparoscopic or robotically assisted
* Any T-stage is eligible (American Joint Committee on Cancer \[AJCC\] 8th edition \[ed\])
* Appropriate stage for study entry based on fluciclovine F-18 PET scan (FACBC, Axumin) F-18 prostate-specific membrane antigen (PSMA) PET (PyLarify) scan, Gallium-68 PSMA PET scan, flotufolastat F-18 PSMA PET scan (Posluma), or C-11 or F-18 Choline PET within 90 days prior to registration that is negative for distant metastatic (M1a, M1b, M1c) disease. For patients with PSA \< 0.20 ng/mL at time of registration, PET scan is recommended but not required
* Pathologically node positive disease with nodal involvement only in the pelvis in the prostatectomy specimen or nodal disease on imaging at time of recurrence (including external iliacs, internal iliacs, and/or obturator nodes); peri-prostatic and peri-rectal nodes can also be considered regional lymphadenopathy and are allowed
* History/physical examination within 90 days prior to registration
* Age \>= 18
* Eastern Cooperative Oncology Group (ECOG) performance status of 0-1 within 90 days prior to registration
* Detectable PSA after radical prostatectomy. Detectable PSA is defined as serum PSA \> 0 ng/mL at least 30 days after prostatectomy
* Patients who have already started on post-prostatectomy GnRH agonist/antagonist for =\< 180 days prior to registration are eligible (Note: patients who started on an oral antiandrogen are eligible if started =\< 180 days and stopped prior to registration)
* Hemoglobin \>= 9.0 g/dL, independent of transfusion and/or growth factors (within 90 days prior to registration)
* Platelet count \>= 100,000 x 10\^9/uL independent of transfusion and/or growth factors (within 90 days prior to registration)
* Serum potassium \>= 3.5 mmol/L within 90 days prior to registration
* Creatinine clearance (CrCl) \>= 30 mL/min estimated by Cockcroft-Gault (please use actual weight for calculation unless greater than 30% above ideal body weight then use the adjusted body weight) (within 90 days prior to registration)
* Total bilirubin =\< 1.5 x institutional upper limit of normal (ULN) (Note: In subjects with Gilbert's syndrome, if total bilirubin is \> 1.5 x ULN, measure direct and indirect bilirubin and if direct bilirubin is =\< 1.5 x ULN, subject is eligible) (within 90 days prior to registration)
* Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\]) or alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2.5 x institutional ULN (within 90 days prior to registration)
* Serum albumin \>= 3.0 g/dL (within 90 days prior to registration)
* Discontinue or substitute concomitant medications known to lower the seizure threshold at least 30 days prior to registration
* The patient must agree to use a condom (even men with vasectomies) and another effective method of birth control if he is having sex with a woman of childbearing potential or agree to use a condom if he is having sex with a woman who is pregnant while on study drug and for 3 months following the last dose of study drug
* Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial and have a CD4 count \>= 200 cells/microliter within 30 days prior to registration. Note: HIV testing is not required for eligibility for this protocol
* For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy within 30 days prior to registration, if indicated. Note: HBV viral testing is not required for eligibility for this protocol
* Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load within 30 days prior to registration
* Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial. Note: Any patient with a cancer (other than keratinocyte carcinoma or carcinoma in situ) who has no evidence of disease for \< 3 years must contact the principal investigator, Ronald Chen, Doctor of Medicine (MD)
* The patient or a legally authorized representative must provide study-specific informed consent prior to study entry
Exclusion Criteria:
* Definitive radiologic evidence of metastatic disease (M1a, M1b or M1c) on molecular imaging (e.g. Fluciclovine F-18 PET, \[FACBC, Axumin\], F-18 PSMA PET \[Pylarify\], flotufolastat F-18 PSMA PET scan \[Posluma\], Gallium-68 PSMA PET scan or C-11 choline PET)
* Prior systemic chemotherapy for the study cancer; note that prior chemotherapy for a different cancer is allowed (completed \> 3 years prior to registration)
* Prior radiotherapy to the region of the study cancer that would result in overlap of radiation therapy fields
* Androgen deprivation therapy (ADT) prior to radical prostatectomy
* Prior treatment with androgen receptor signaling inhibitor (including but not exclusive to a growing list of: abiraterone acetate, enzalutamide, apalutamide, darolutamide), unless started =\< 180 days and stopped prior to registration, which is allowed
* Current use of 5-alpha reductase inhibitor. NOTE: if the alpha reductase inhibitor is stopped prior to randomization the patient is eligible
* History of any of the following:
* Seizure or known condition that may pre-dispose to seizure (e.g. prior stroke within 1 year prior to registration, brain arteriovenous malformation, Schwannoma, meningioma, or other benign central nervous system \[CNS\] or meningeal disease which may require treatment with surgery or radiation therapy)
* Severe or unstable angina, myocardial infarction, arterial or venous thromboembolic events (e.g., pulmonary embolism, cerebrovascular accident including transient ischemic attacks), or clinically significant ventricular arrhythmias within 12 months prior to registration
* New York Heart Association functional classification III/IV (Note: Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association functional classification.)
* History of any condition that in the opinion of the investigator, would preclude participation in this study
* Current evidence of any of the following:
* Known gastrointestinal disorder affecting absorption of oral medications
* Active uncontrolled infection
* Presence of uncontrolled hypertension (persistent systolic blood pressure \[BP\] \>= 160 mmHg or diastolic BP \>= 100 mmHg). Subjects with a history of hypertension are allowed, provided that BP is controlled to within these limits by anti-hypertensive treatment
* Any chronic medical condition requiring a higher dose of corticosteroid than 10 mg prednisone/prednisolone once daily
* Baseline moderate and severe hepatic impairment (Child-Pugh Class B \& C)
* Inability to swallow oral pills
* Any current condition that in the opinion of the investigator, would preclude participation in this study
* Patients must not plan to participate in any other therapeutic clinical trials while receiving treatment on this study
* Patients with inflammatory bowel disease DRUG: Apalutamide, PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Scan, PROCEDURE: Computed Tomography, DRUG: Hormone Therapy, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Positron Emission Tomography, OTHER: Quality-of-Life Assessment, OTHER: Questionnaire Administration, RADIATION: Radiation Therapy
Prostate Adenocarcinoma, Stage I Prostate Cancer AJCC v8, Stage II Prostate Cancer AJCC v8, Stage III Prostate Cancer AJCC v8, Stage IVA Prostate Cancer AJCC v8
Prostate Cancer, Apalutamide, Abiraterone Acetate
Endovascular Ablation of the Right Greater Splanchnic Nerve in Subjects Having HFpEF (Rebalance-HF)
Judit Adorjan - j.adorjan@axontherapies.com
Shah, Keyur, B
NCT04592445
HM20022228
Inclusion Criteria
• Chronic heart failure, defined as:
• Symptoms of HF requiring current (intermittent or continuous) treatment with diuretics for \>30 days, AND
• NYHA class II with a history of \>NYHA class II in the past year, NYHA class III, or ambulatory NYHA class IV symptoms (paroxysmal nocturnal dyspnea, orthopnea, dyspnea on mild or moderate exertion) at screening or signs of HF (any rales post cough, chest x-ray demonstrating pulmonary congestion), AND
• At least one of the following: i. ≥1 HF hospital admission (with HF as the primary diagnosis) including treatment with intravenous (IV) diuretics or urgent unplanned treatment with IV diuretics in healthcare facility within past 12 months, OR ii. NT-proBNP \>300 pg/ml in normal sinus rhythm (\>450 pg/ml in atrial fibrillation or flutter) within the past 6 months; BNP \>100 pg/ml in normal sinus rhythm (\>300 pg/ml in atrial fibrillation or flutter) within the past 6 months, OR iii. Right heart catheterization (RHC) with PCWP ≥ with PCWP ≥18 mmHg at rest or 25 mmHg during exercise at the time of the screening RHC.
• Ongoing stable GDMT HF management (unless unable to tolerate GDMT) and management of potential comorbidities according to the 2022 ACCF/AHA Guideline for the Management of Heart Failure (Class 1 and 2a recommendations), with no significant changes \[≥100% increase or ≥50% decrease\] for a minimum of 1 month (30 days) prior to screening, that is expected to be maintained without change for at least 6 months. Participants cannot have started a glucagon-like peptide (GLP)-1 or gastric inhibitory peptide (GIP) agonist within the last 6 months or plan to start a GLP-1 or GIP agonist within the ensuing 6 months after enrollment.
• LVEF ≥50% (site-determined by transthoracic echocardiography) within the past 6 months.
• Age ≥40 years.
• Subject is willing and able to provide appropriate study-specific informed consent, follow protocol procedures, and comply with follow-up visit requirements.
• MI (type I) and/or percutaneous cardiac intervention within 3 months prior to screening; CABG in past 3 months prior to screening, or current indication for coronary revascularization.
• Cardiac resynchronization therapy initiated within 3 months prior to screening.
• Advanced heart failure defined as one or more of the following:
• ACC/AHA/ESC Stage D HF or non-ambulatory NYHA Class IV HF.
• Inotropic infusion (continuous or intermittent) within 6 months prior to screening.
• Subject is on the cardiac transplant waiting list.
• Presence of or prior history of mechanical circulatory support for HF.
• Poor left heart compliance as determined by pulse-wave Doppler transmitral early-to-late (E/A) ratio \>2.0 assessed by the screening echocardiogram. The Screening Committee will evaluate left heart function if the transmitral A velocity is not measurable or absent.
• Right heart dysfunction defined as tricuspid annular plane systolic excursion (TAPSE) \<12 mm or right ventricular (RV) fractional area change (FAC) \<25% assessed by the screening echocardiogram.
• Body mass index (BMI) \>45 kg/m2.
• 6-minute walk test distance \<100 meters OR \>450 meters.
• Admission for HF within the 30 days prior to planned index procedure.
• Any known history of orthostatic hypotension or orthostatic hypotension at the time of screening (regardless of the presence of symptoms). Orthostatic hypotension is defined as a systolic blood pressure (BP) decrease of \>20 mmHg upon going from supine to standing position or undergoing treatment with Midodrine.
• Orthostatic pulse pressure change from supine to standing decrease of \>10mmHg in the absence of a HR increase \>15bpm
• Postural orthostatic tachycardia syndrome or preload insufficiency syndrome.
• Systolic BP \<100 mmHg or \>170 mmHg despite appropriate medical management.
• Baseline screening ECG resting HR \>100 beats per minute or ventricular tachycardia.
• Catheter ablation for atrial fibrillation within 6 months prior to screening or planned in the next 12 months at the time of screening.
• Left ventricular EF \<40% within the 3 years prior to screening unless reduced EF was transient and associated with an acute event.
• Presence of significant valve disease defined by the site cardiologist as:
• Greater than mild mitral valve stenosis.
• Greater than moderate mitral valve regurgitation.
• Greater than moderate-to-severe tricuspid valve regurgitation.
• Greater than moderate aortic valve stenosis or regurgitation.
• Known hypertrophic cardiomyopathy, restrictive cardiomyopathy, constrictive pericarditis, cardiac amyloidosis, or other infiltrative cardiomyopathy (e.g., hemochromatosis, sarcoidosis).
• History of clinically significant liver cirrhosis.
• Prior weight loss surgery
• Dialysis dependent; or estimated GFR \<25 ml/min/1.73 m2 by CKD-EPI creatinine equation.
• Arterial oxygen saturation \<90% on room air.
• Chronic pulmonary disease requiring continuous home oxygen OR hospitalization for exacerbation of chronic pulmonary disease (including intubation) in the 12 months before study entry OR known history of GOLD Class III or worse chronic obstructive pulmonary disease (COPD).
• Participating in conflicting investigational drug or device study that is not completed within 30 days prior to the screening visit.
• Life expectancy \<12 months for non-cardiovascular reasons.
• Any condition, or history of illness or surgery that, in the opinion of the site investigator or Screening Committee, might confound the results of the study or pose additional risks to the patient.
• Females who are pregnant or lactating or planning to become pregnant during the next year.
• Any of the following measured by screening right heart catheterization:
• Mean right atrial pressure (RAP) \>20 mmHg at rest
• Cardiac index \<2.0 L/min/m2 at rest
• Pulmonary vascular resistance (PVR) \>4 Wood units Exclusion Criteria Assessed During the index procedure:
• Vessel tortuosity or variant vascular anatomy that could preclude the access or maneuvering of the interventional device from the access site to target vessel. This includes previous spine surgery that may impact the ability to access and treat the target sites of T11 and T10.
• Chronic heart failure, defined as:
• Symptoms of HF requiring current (intermittent or continuous) treatment with diuretics for \>30 days, AND
• NYHA class II with a history of \>NYHA class II in the past year, NYHA class III, or ambulatory NYHA class IV symptoms (paroxysmal nocturnal dyspnea, orthopnea, dyspnea on mild or moderate exertion) at screening or signs of HF (any rales post cough, chest x-ray demonstrating pulmonary congestion), AND
• At least one of the following: i. ≥1 HF hospital admission (with HF as the primary diagnosis) including treatment with intravenous (IV) diuretics or urgent unplanned treatment with IV diuretics in healthcare facility within past 12 months, OR ii. NT-proBNP \>300 pg/ml in normal sinus rhythm (\>450 pg/ml in atrial fibrillation or flutter) within the past 6 months; BNP \>100 pg/ml in normal sinus rhythm (\>300 pg/ml in atrial fibrillation or flutter) within the past 6 months, OR iii. Right heart catheterization (RHC) with PCWP ≥ with PCWP ≥18 mmHg at rest or 25 mmHg during exercise at the time of the screening RHC.
• Ongoing stable GDMT HF management (unless unable to tolerate GDMT) and management of potential comorbidities according to the 2022 ACCF/AHA Guideline for the Management of Heart Failure (Class 1 and 2a recommendations), with no significant changes \[≥100% increase or ≥50% decrease\] for a minimum of 1 month (30 days) prior to screening, that is expected to be maintained without change for at least 6 months. Participants cannot have started a glucagon-like peptide (GLP)-1 or gastric inhibitory peptide (GIP) agonist within the last 6 months or plan to start a GLP-1 or GIP agonist within the ensuing 6 months after enrollment.
• LVEF ≥50% (site-determined by transthoracic echocardiography) within the past 6 months.
• Age ≥40 years.
• Subject is willing and able to provide appropriate study-specific informed consent, follow protocol procedures, and comply with follow-up visit requirements.
Exclusion Criteria:
• MI (type I) and/or percutaneous cardiac intervention within 3 months prior to screening; CABG in past 3 months prior to screening, or current indication for coronary revascularization.
• Cardiac resynchronization therapy initiated within 3 months prior to screening.
• Advanced heart failure defined as one or more of the following:
• ACC/AHA/ESC Stage D HF or non-ambulatory NYHA Class IV HF.
• Inotropic infusion (continuous or intermittent) within 6 months prior to screening.
• Subject is on the cardiac transplant waiting list.
• Presence of or prior history of mechanical circulatory support for HF.
• Poor left heart compliance as determined by pulse-wave Doppler transmitral early-to-late (E/A) ratio \>2.0 assessed by the screening echocardiogram. The Screening Committee will evaluate left heart function if the transmitral A velocity is not measurable or absent.
• Right heart dysfunction defined as tricuspid annular plane systolic excursion (TAPSE) \<12 mm or right ventricular (RV) fractional area change (FAC) \<25% assessed by the screening echocardiogram.
• Body mass index (BMI) \>45 kg/m2.
• 6-minute walk test distance \<100 meters OR \>450 meters.
• Admission for HF within the 30 days prior to planned index procedure.
• Any known history of orthostatic hypotension or orthostatic hypotension at the time of screening (regardless of the presence of symptoms). Orthostatic hypotension is defined as a systolic blood pressure (BP) decrease of \>20 mmHg upon going from supine to standing position or undergoing treatment with Midodrine.
• Orthostatic pulse pressure change from supine to standing decrease of \>10mmHg in the absence of a HR increase \>15bpm
• Postural orthostatic tachycardia syndrome or preload insufficiency syndrome.
• Systolic BP \<100 mmHg or \>170 mmHg despite appropriate medical management.
• Baseline screening ECG resting HR \>100 beats per minute or ventricular tachycardia.
• Catheter ablation for atrial fibrillation within 6 months prior to screening or planned in the next 12 months at the time of screening.
• Left ventricular EF \<40% within the 3 years prior to screening unless reduced EF was transient and associated with an acute event.
• Presence of significant valve disease defined by the site cardiologist as:
• Greater than mild mitral valve stenosis.
• Greater than moderate mitral valve regurgitation.
• Greater than moderate-to-severe tricuspid valve regurgitation.
• Greater than moderate aortic valve stenosis or regurgitation.
• Known hypertrophic cardiomyopathy, restrictive cardiomyopathy, constrictive pericarditis, cardiac amyloidosis, or other infiltrative cardiomyopathy (e.g., hemochromatosis, sarcoidosis).
• History of clinically significant liver cirrhosis.
• Prior weight loss surgery
• Dialysis dependent; or estimated GFR \<25 ml/min/1.73 m2 by CKD-EPI creatinine equation.
• Arterial oxygen saturation \<90% on room air.
• Chronic pulmonary disease requiring continuous home oxygen OR hospitalization for exacerbation of chronic pulmonary disease (including intubation) in the 12 months before study entry OR known history of GOLD Class III or worse chronic obstructive pulmonary disease (COPD).
• Participating in conflicting investigational drug or device study that is not completed within 30 days prior to the screening visit.
• Life expectancy \<12 months for non-cardiovascular reasons.
• Any condition, or history of illness or surgery that, in the opinion of the site investigator or Screening Committee, might confound the results of the study or pose additional risks to the patient.
• Females who are pregnant or lactating or planning to become pregnant during the next year.
• Any of the following measured by screening right heart catheterization:
• Mean right atrial pressure (RAP) \>20 mmHg at rest
• Cardiac index \<2.0 L/min/m2 at rest
• Pulmonary vascular resistance (PVR) \>4 Wood units Exclusion Criteria Assessed During the index procedure:
• Vessel tortuosity or variant vascular anatomy that could preclude the access or maneuvering of the interventional device from the access site to target vessel. This includes previous spine surgery that may impact the ability to access and treat the target sites of T11 and T10.
DEVICE: Greater Splanchnic Nerve Ablation, DEVICE: Sham Control
Heart Failure With Preserved Ejection Fraction (HFpEF
Heart Failure with Preserved Ejection Fraction (HFpEF), HFpEF, Greater Splanchnic Nerve, GSN
Preventing Firearm Violence in Youth: A Hospital-based Prevention Strategy
Nicholas Thomson - Nicholas.Thomson@vcuhealth.org
Thomson, Nicholas
NCT05078164
HM20022975
Inclusion Criteria:
• Youth are aged 10-17 years and their adult caregivers are aged 18 years and older
• Receiving treatment in the hospital for a violence-related injury (e.g., gunshot wound) or referred to BTG/IVPP services
• English speaking
• Eligible for BTG services (which includes living within the BTG catchment area for the hospital; Richmond City and neighboring counties)
Exclusion Criteria:
• Youth are \< 10 years old
• Youth are \> 18 years old
• Prisoners
BEHAVIORAL: Bridging the Gap (BTG)
Violence in Adolescence
CONTIGO - A Narrative Intervention to Enhance Genetic Counseling and Testing
Alejandra Hurtado de Mendoza, Ph.D - ahd28@georgetown.edu
NCT05130606
Inclusion Criteria:
Aim 1 and 2.
* Self-identify as a Latina woman
* Be 18 years old or older
* Be able to provide informed consent
* Be fluent in Spanish
* Meet NCCN criteria to be considered for genetic cancer risk assessment for HBOC, whether by a personal history of cancer or family history of cancer
* No previous participation in genetic counseling or testing for hereditary breast and ovarian cancer risk
* No other family members are participating in this study
* Have not participated in any previous studies involving interventions about HBOC or GCT
Aim 3.
* Be 18 years old or older
* Be fluent in English or Spanish
* Have a role in the partner community clinic as either a) full-time or part-time employee b) intern c) volunteer
Exclusion Criteria:
\- BEHAVIORAL: Culturally Targeted Narrative Video: "Is My Cancer Hereditary? Rosa Visits a Genetic Counselor.", BEHAVIORAL: FORCE Fact Sheet
Hereditary Breast and Ovarian Cancer
Breast cancer, Ovarian cancer, Hereditary cancer, Latinas
MYTHS - MYocarditis THerapy With Steroids (MYTHS)
Enrico Ammirati, MD, PhD - enrico.ammirati@ospedaleniguarda.it
NCT05150704
Inclusion Criteria:
* Patients admitted to hospital for suspected AM
* Age 18 years or older and below 70 years (18-69 years)
* Acute HF with clinically suspected acute myocarditis based on an N-terminal pro-B-type natriuretic peptide (NT-proBNP) concentration of 1600 pg/mL or more or a B-type natriuretic peptide (BNP) concentration of 400 pg/mL or more;
* Left ventricular ejection fraction (LVEF)\<41% and left ventricular end diastolic diameter (LV-EDD)\<56 mm (parasternal long-axis view) on echocardiogram;
* Increased troponin (3x upper reference limit \[URL\]) at the time of randomization;
* Clinical onset of cardiac symptoms within 3 weeks from randomization;
* Excluded coronary artery disease by coronary angiogram in subjects ≥46 years of age, in case myocarditis is not histologically proven;
* Randomization within 120 hours from hospital admission.
Exclusion Criteria:
* Known systemic autoimmune disorder or other conditions at the time of randomization where immunosuppression is assumed useful. Patients in whom a systemic autoimmune disorder will be diagnosed during hospitalization will be included in the study if randomized, including patients with a diagnosis of cardiac sarcoidosis or giant cell myocarditis (GCM). Both patients included in the corticosteroids-treatment arm or in the placebo-treatment arm can receive the standard immunosuppressive therapy used in the center since the diagnosis of a systemic autoimmune disorder, or cardiac sarcoidosis or GCM;
* Patients already on oral/IV chronic corticosteroid therapy or other chronic immunosuppressive therapies (colchicine or nonsteroidal anti-inflammatory drugs \[NSAIDs\] are not considered immunosuppressive drugs);
* Contraindication to corticosteroids, including allergies to this medication and its excipients;
* Patients with persistent peripheral eosinophilia (persistent Eosinophil count \>7% of the leukocytes) or known hypereosinophilic syndrome at the time of randomization. Patients in whom eosinophilic myocarditis will be diagnosed on endomyocardial biopsy (EMB) will be included in the study if already randomized. Both patients included in the corticosteroids-treatment arm or in the placebo-treatment arm can receive the standard immunosuppressive therapy used in the center since the diagnosis;
* Myocarditis associated with the ongoing administration of anti-cancer immune checkpoint inhibitor (ICI) agents;
* Previously known chronic cardiac disease (i.e., previous cardiomyopathy) that does NOT include previous myocarditis if there is a functional recovery at the time of screening);
* Evidence of active bacterial or fungal infectious disease (presence of fever or increased C-reactive protein are not considered exclusion criteria), or suspected bacterial/fungal infection associated with increased levels of procalcitonin (cut-off \>10 ng/mL), if the laboratory exam is available in the center;
* Known chronic infective disease, such as HIV infection or tuberculosis;
* out-of-hospital cardiac arrest;
* t-MCS instituted more than 48 hours before randomization;
* Patients clinically judged too sick to initiate t-MCS (i.e., irreversible multiorgan failure);
* Echocardiographic presence of images suggestive of other cardiac diseases (i.e. endocarditis)
* Participants involved in another clinical trial;
* Pregnant women (known pregnancy) or POSITIVE human chorionic gonadotropin (HCG) test measures (urine/blood) for women of 18-50 years of age.
* Any other significant disease with expected life expectancy \<12 months (i.e., evidence of irreversible severe brain injury) or disorder which, in the opinion of the Investigator, may either put the participants at risk because of participation in the trial, or may influence the result of the trial, or the participant's ability to participate in the trial. DRUG: Methylprednisolone, DRUG: saline solution
Myocarditis Acute
Acute Myocarditis, Corticosteroid therapy, Myocarditis, Trial, Immunosuppression, Acute heart failure, Fulminant acute myocarditis
Comparing the Clinical Impact of Pancreatic Cyst Surveillance Programs and Associated Biomarkers
Dickens, Ariana - dickensa5@vcu.edu
Trevino, Jose
NCT04239573
HM20020533
Inclusion Criteria:
* Patient must be ≥ 50 years and ≤ 75 years of age
* Patient must not have acute pancreatitis or a history of chronic pancreatitis
* Patient must have received a CT, MRI, or EUS within 6 months prior to enrollment that revealed one or more ≥ 1 cm pancreatic cyst(s).
* Patients of childbearing potential must not be known to be pregnant
* Patient must not have a prior diagnosis of pancreatic malignancy of any type
* Patient must not have a history of pancreatic resection
* Patients with only pancreatic lesions without malignant risk (pancreatic pseudocyst or classic serous cystic lesion) are not eligible
* Patient must not have a family history of pancreatic adenocarcinoma in one or more first-degree relatives (biological parents, full siblings or children)
* Patient must not have pancreatic cyst morphology that would prompt immediate surgical consideration (enhancing mural nodule, solid component in cyst, pancreatic duct ≥ 10mm, cyst causing obstructive jaundice)
* Patient must not have a comorbid illness that precludes EUS or pancreatic cyst resection
* Patient must not be in any form of pancreatic cyst surveillance for \> 1 year, defined as organized, periodic up-to-date imaging directed towards the pancreatic cyst of interest
* PRIOR TO ADDENDUM #5 08/13/2024: Patient must be ≥ 50 years and ≤ 75 years of age
* PRIOR TO ADDENDUM #5 08/13/2024: Patient must not have acute pancreatitis or a history of chronic pancreatitis.
* PRIOR TO ADDENDUM #5 08/13/2024: Patient must have received a CT, MRI, or EUS within 6 months prior to randomization that revealed one or more ≥ 1 cm pancreatic cyst (s).
* PRIOR TO ADDENDUM #5 08/13/2024: Patients of childbearing potential must not be known to be pregnant.
* PRIOR TO ADDENDUM #5 08/13/2024: Patient must not have a prior diagnosis of pancreatic malignancy of any type.
* PRIOR TO ADDENDUM #5 08/13/2024: Patient must not have a history of pancreatic resection.
* PRIOR TO ADDENDUM #5 08/13/2024: Patients with only pancreatic lesions without malignant risk (pancreatic pseudocyst or classic serous cystic lesion) are not eligible.
* PRIOR TO ADDENDUM #5 08/13/2024: Patient must not have a family history of pancreatic adenocarcinoma in one or more first-degree relatives (biological parents, full siblings or children).
* PRIOR TO ADDENDUM #5 08/13/2024: Patient must not have pancreatic cyst morphology that would prompt immediate surgical consideration (enhancing mural nodule, solid component in cyst, pancreatic duct ≥10mm, cyst causing obstructive jaundice).
* PRIOR TO ADDENDUM #5 08/13/2024: Patient must not have a comorbid illness that precludes EUS or pancreatic cyst resection. PROCEDURE: Biopsy Procedure, PROCEDURE: Biospecimen Collection, PROCEDURE: Computed Tomography, PROCEDURE: Endoscopic Ultrasound, PROCEDURE: Endoscopic Ultrasound-Guided Fine-Needle Aspiration, PROCEDURE: Magnetic Resonance Imaging, OTHER: Quality-of-Life Assessment, OTHER: Questionnaire Administration, PROCEDURE: Surgical Procedure
Pancreatic Neoplasm
Treating Heart Dysfunction Related to Cancer Therapy With Sacubitril/Valsartan (TREAT-HF)
Massey CTO CPC Team - masseycpc@vcu.edu
Bottinor, Wendy
NCT05194111
HM20023601
Inclusion Criteria:
* Age \/=40% on echocardiogram or cardiac MRI
* No symptoms of heart failure (shortness of breath, fatigue, swelling). Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea (shortness of breath).
Exclusion Criteria:
* -Age \<18 years
* Inability to obtain consent from patient or legally authorized representative
* Active acute or chronic psychiatric illness that in the opinion of the investigator may prevent compliance with study instructions
* Limited English or Spanish proficiency that in the opinion of the investigator may prevent understanding the content of the informed consent form or safely completing the study procedures
* Participation in another concurrent intervention study within 30 days or treatment with an investigational drug within 5 half-lives prior to randomization
* Greater than mild mitral or aortic valve regurgitation/stenosis known pre-cancer therapy
* Severe kidney disease (GFR \<30 mL/min/1.73m2)
* Chronic hyperkalemia (\>5mmol/L)
* Evidence of COVID-19 within the last 60 days or recent (21 days) exposure to close personal contact with COVID-19.
* Greater than moderate tricuspid or pulmonary valve regurgitation/stenosis known pre-cancer therapy
* Hemodynamically significant congenital heart disease in the opinion of the investigator (not including PFO/small ASD or small VSD)
* Greater than moderate pericardial effusion
* Constrictive cardiomyopathy diagnosed pre-cancer therapy
* Family history of genetic cardiomyopathy
* Evidence of infiltrative cardiomyopathy
* Symptomatic heart disease based on NYHA classification
* Allergy to valsartan or sacubitril
* Inability to complete CMR or 6-minute walk test
* Inability to measure non-invasive blood pressure and heart rate in the ambulatory/home setting
* Pregnant/lactating
* History of severe hypersensitivity reactions to gadolinium-based contrast agents (will perform limited cardiac imaging without contrast)
* Concomitant use of other ACE inhibitors or ARBs, aliskiren, NSAIDs or lithium or the inability to stop these medications for the study DRUG: Sacubitril-valsartan, DRUG: Valsartan
Heart Failure, Heart Dysfunction
DMCRN-02-001: Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)
Ruby Langeslay - ruby.langeslay@vcuhealth.org
Johnson, Nicholas, E
NCT05224778
HM20023386
Inclusion Criteria:
* Age neonate to 3 years 11 months at enrollment.
* A diagnosis of CDM, which is defined as children having symptoms of myotonic dystrophy in the newborn period (\<30 days), such as hypotonia, feeding or respiratory difficulty, requiring hospitalization to a ward or to the neonatal intensive care unit for more than 72 hours; and a genetic test confirming an expanded trinucleotide (CTG) repeat in the DMPK gene in the child or mother. An expanded CTG repeat size in the child is considered greater than 200 repeats or E1-E4 classification (E1= 200-500, E2=500-1,000, E3=1,000-1,500, E4\>1,500).
* Guardian is willing and able to sign consent and follow study procedures
Exclusion Criteria:
* Any other non-DM1 illness that would interfere with the ability or results of the study in the opinion of the site investigator
* Significant trauma within one month
* Internal metal or devices (exclusion for DEXA component)
* History of bleeding disorder or platelet count \<50,000
* History of reaction to local anesthetic Congenital Myotonic Dystrophy, CDM
Clinical Research, Myotonic dystrophy, Congenital Myotonic Dystrophy, CDM
A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT
Gwaltney, Lindsey - lbgwaltney@vcu.edu
Austin, Frances
NCT04322318
HM20023079
Inclusion Criteria:
* Patients with newly diagnosed stages 2 - 4 diffuse anaplastic Wilms tumor must be enrolled on APEC14B1, consented to Part A - Eligibility Screening, and have received an initial stratum assignment showing DAWT (if anaplasia first identified at diagnostic, pre-treatment nephrectomy or biopsy) or a final stratum assignment showing DAWT (if anaplasia first noted at delayed nephrectomy) prior to enrollment on AREN1921. Prior enrollment on APEC14B1 is not an eligibility requirement for patients with relapsed favorable histology Wilms tumor.
* Patients must be =\< 30 years old at study enrollment
* Patients with the following diagnoses are eligible for this study:
* Newly diagnosed stages 2 - 4 diffuse anaplastic Wilms tumor as confirmed by central review
* Favorable histology Wilms tumor at first relapse. Relapsed FHWT patients must have previously achieved remission for their initial FHWT diagnosis to be eligible for this study. The relapse risk groups are defined as follows, regardless of radiation therapy:
* Standard-Risk relapse: Patients who received two chemotherapy agents for frontline therapy; primarily actinomycin D and vincristine
* High-Risk relapse: Patients who received three chemotherapy agents for frontline therapy; primarily vincristine, actinomycin D and doxorubicin or vincristine, actinomycin D and irinotecan
* Very High-Risk relapse: Patients who received four or more chemotherapy agents as part of initial therapy; primarily regimen M or its variations
* Patients with newly diagnosed DAWT must have had histologic verification of the malignancy. For relapsed FHWT patients, biopsy to prove recurrence is encouraged, but not required
* Note: For relapsed FHWT patients, an institutional pathology report confirming favorable histology Wilms tumor (from relapse, if available, or from original diagnosis) must be available for upload prior to initiation of protocol therapy
* Patients with newly diagnosed Stages 2 - 4 diffuse anaplastic Wilms tumor must be enrolled on AREN1921 within 2 weeks of the tumor-directed surgery or biopsy procedure that first confirms a diagnosis of DAWT, whether at initial diagnostic procedure or delayed nephrectomy (such surgery/biopsy is day 0). For patients who received prior therapy for presumed favorable histology Wilms tumor, later confirmed to have diffuse anaplastic Wilms tumor at subsequent review of the initial biopsy
* Patients with newly diagnosed DAWT who undergo upfront nephrectomy must have at least 1 lymph node sampled prior to study enrollment
* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age
* Patients must have a life expectancy of \>= 8 weeks
* Diffuse Anaplastic Wilms Tumor: Patients with diffuse anaplastic histology must have had no prior systemic therapy, except in the following situations:
* Patients with diffuse anaplastic Wilms tumor who received no more than 12 weeks of pre nephrectomy chemotherapy for what was originally presumed to be favorable histology Wilms tumor, subsequently confirmed to be diffuse anaplastic Wilms tumor at delayed nephrectomy
* Patients with diffuse anaplastic Wilms tumor who received no more than 6 weeks of chemotherapy following upfront biopsy, initiated within 14 days of biopsy, for presumed favorable histology Wilms tumor based on institutional review, but subsequently corrected to diffuse anaplastic Wilms tumor based on the initial stratum assignment on APEC14B1-REN
* Treatment consisting of vincristine/doxorubicin/cyclophosphamide initiated on an emergent basis and within allowed timing as described
* Note: Patients who received prior therapy for presumed favorable histology Wilms tumor, later identified to have diffuse anaplastic Wilms tumor as per above, must begin study treatment starting at cycle 3 (week 7) of regimen UH 3. Patients who received emergency radiation to preserve organ function are eligible as noted. Patients who received radiation as part of standard of care for presumed newly diagnosed favorable histology Wilms tumor, along with chemotherapy as noted above, prior to identification of diffuse anaplasia, are also eligible
* Relapsed Favorable Histology Wilms Tumor: Patients must not have received prior chemotherapy for their relapsed favorable histology Wilms tumor diagnosis. In addition, patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study
* Myelosuppressive chemotherapy: Must not have received within 2 weeks of entry onto this study
* Radiation therapy (RT): \>= 2 weeks (wks) must have elapsed for local palliative RT (small port); \>= 6 months must have elapsed if prior craniospinal RT or if \>= 50% radiation of pelvis; \>= 6 wks must have elapsed if other substantial bone marrow (BM) radiation. Patients with relapsed favorable histology Wilms tumor who received emergency radiation to preserve organ function are eligible and do not need to washout with the above criteria
* Patients may not be receiving any other investigational agents (within 4 weeks prior to study enrollment)
* Peripheral absolute neutrophil count (ANC) \>= 750/uL (performed within 7 days prior to enrollment)
* Platelet count \>= 75,000/uL (transfusion independent) (performed within 7 days prior to enrollment)
* Hemoglobin \>= 8.0 g/dL (may receive red blood cell \[RBC\] transfusions) (performed within 7 days prior to enrollment)
* Patients with high-risk or very high-risk relapsed FHWT who will be treated with regimen ICE/Cyclo/Topo, must have renal function assessed by creatinine clearance or radioisotope glomerular filtration rate (GFR) and meet the following requirement:
* Creatinine clearance or radioisotope GFR \>= 60 mL/min/1.73 m\^2 (performed within 7 days prior to enrollment)
* Patients diagnosed with stage 2-4 DAWT or standard risk relapsed FHWT, who will be treated with regimen UH 3, may either obtain a creatinine clearance, radioisotope GFR (meeting the above criteria of GFR \>= 60 mL/min/1.73 m\^2), or an adequate serum creatinine as per the following table:
* Age: Maximum Serum Creatinine (mg/dL)
* 1 month to \< 6 months: 0.4 (male and female)
* 6 months to \< 1 year: 0.5 (male and female)
* 1 to \< 2 years: 0.6 (male and female)
* 2 to \< 6 years: 0.8 (male and female)
* 6 to \< 10 years: 1 (male and female)
* 10 to \< 13 years: 1.2 (male and female)
* 13 to \< 16 years: 1.5 (male), 1.4 (female)
* \>= 16 years: 1.7 (male), 1.4 (female)
* Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age or direct bilirubin =\< ULN for patients whose total bilirubin \> 1.5 x ULN (performed within 7 days prior to enrollment)
* Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase \[AST\]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 2.5 x upper limit of normal (ULN) for age or =\< 5 x ULN for patients with liver metastases (performed within 7 days prior to enrollment)
* Shortening fraction of \>= 27% by echocardiogram, or ejection fraction of \>= 50% by radionuclide angiogram (obtained within 21 days prior to enrollment and start of protocol therapy)
Exclusion Criteria:
* Patients with a history of bilateral Wilms tumor (synchronous or metachronous)
* Patients with any uncontrolled, intercurrent illness including, but not limited to, ongoing or active infection, or symptomatic congestive heart failure (defined as grade 2 or higher heart failure per Common Terminology Criteria for Adverse Events \[CTCAE\] version 5.0)
* Relapsed FHWT patients who did not receive frontline chemotherapy (e.g., very low risk FHWT initially observed without chemotherapy) or received only one chemotherapy agent for frontline therapy
* For patients with high-risk or very high-risk relapsed FHWT:
* Patients with renal tubular acidosis (RTA) as evidenced by serum bicarbonate \< 16 mmol/L and serum phosphate =\< 2 mg/dL (or \< 0.8 mmol/L) without supplementation
* For stages 2-4 DAWT and standard-risk relapsed FHWT patients:
* Chronic inflammatory bowel disease and/or bowel obstruction
* Concomitant use of St. John's wort, which cannot be stopped prior to the start of trial treatment
* Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
* Lactating females who plan to breastfeed their infants
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation PROCEDURE: Biopsy Procedure, PROCEDURE: Biospecimen Collection, PROCEDURE: Bone Scan, DRUG: Carboplatin, PROCEDURE: Computed Tomography, DRUG: Cyclophosphamide, DRUG: Doxorubicin, DRUG: Etoposide, DRUG: Ifosfamide, DRUG: Irinotecan, PROCEDURE: Magnetic Resonance Imaging, PROCEDURE: Positron Emission Tomography, RADIATION: Radiation Therapy, PROCEDURE: Surgical Procedure, DRUG: Topotecan, PROCEDURE: Transabdominal Ultrasound, DRUG: Vincristine, PROCEDURE: X-Ray Imaging
Anaplastic Kidney Wilms Tumor, Recurrent Kidney Wilms Tumor, Stage II Kidney Wilms Tumor, Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor
An Internet-based Program to Help Cancer Survivors Manage Pain (IMPACTS)
Karen Craver - NCORP@wakehealth.edu
Ameringer, Suzanne, W.
NCT04462302
HM20022887
Inclusion Criteria:
Must have a documented diagnosis of invasive cancer that has been treated with either single modality therapy or any combination of surgery, radiation, and chemotherapy/drug therapy (e.g. cytotoxic therapy, targeted therapy, immunotherapy, hormonal therapy, etc.). Patients with a cancer history of only superficial skin cancers or in situ malignancy are not eligible.
* May be either off all treatment OR actively receiving anticancer therapy in an adjuvant setting, maintenance setting, or for active cancer.
* Patients who currently undergoing anticancer therapy should not have any plans to change or adjust their treatment during the intervention period. This includes changing to another therapy or ending therapy entirely.
* Patients who are currently receiving anticancer therapy at the screening process must have been on current therapy for at least four weeks. Alternatively, if they are planning to discontinue therapy before enrolling, they must have been off therapy for four weeks prior to enrollment.
* A minimum of four weeks must have elapsed since the most recent MAJOR surgical intervention.
* A minimum of two weeks must have elapsed since the most recent MINOR surgical procedure (e.g., port placement).
* In addition, eligible patients must not have a planned surgical procedure or course of radiation therapy during the 3-month study intervention period (i.e., the three months leading up to primary outcome evaluation-timepoint.
* Patients who are no longer receiving anticancer therapy must be less than/equal to 5 years since the completion of their anticancer therapy (e.g., time since the last day of chemotherapy administration, time since last day of radiotherapy, etc.).
* Must have pain indicated by a score of ≥ 4 on PROMIS Pain Intensity (1a) scale, using the Pain Eligibility Interview.
* Must have a score of "Most Days" or higher on the Graded Chronic Pain Scale Revised (Abbreviated) using the Pain Eligibility Interview.
* Patients do not have to be on analgesic medications of any kind in order to participate. If they are taking analgesics, they must be on a stable analgesic regimen (i.e., no changes to the prescribed analgesic regimen) over a period of at least 14 days prior to enrollment. Eligible patients should not have planned upward dose titration of their analgesics during the 3-month study intervention period (i.e., the three months leading up to primary outcome evaluation timepoint. Patients may elect to decrease their analgesic use during the study as per discussions with their provider. Unexpected dose adjustments including dose escalations as a result of unforeseen clinical need is allowed in all patients at all times during the study. Cannabis prescribed for medicinal purposes would qualify as an analgesic in this context.
* Must have pain of new onset or significantly exacerbated since the time of cancer diagnosis or initiation of cancer treatment
* Must be expected to be able to complete all study activities including the 22- and 34-week follow-up assessments according to the treating/referring clinician (e.g., treating clinician feels the patient is unlikely to develop progressive disease requiring additional active cancer therapy through the 6-month follow-up period).
* ECOG performance status of 0, 1, or 2.
* Age ≥18 years at the time of study entry
* Must be able to speak, read and understand English.
Exclusion Criteria:
* Has a disability that precludes completion of study activities (e.g., severe vision or hearing impairment, diagnosis of dementia or clinical evidence of severe cognitive impairment, diagnosis or clinical evidence of severe psychiatric disorder, or diagnosed drug or alcohol abuse disorder), as per patient report or documented in the medical record.
* Reports only preexisting pain conditions unrelated to cancer or cancer treatment (e.g., migraine or tension headache, arthritis, back disorders, bursitis/tendonitis, injuries, fibromyalgia).
* Has a known or suspected diagnosable substance use disorder or opioid overuse disorder (according to DSM-5 criteria), or is actively receiving treatment for a substance use disorder, as per patient report or documented in the medical record.
* Currently being prescribed buprenorphine or suboxone.
* Patients enrolled on hospice care or end-of-life palliative care are not eligible for enrollment. Patients whose local care network provides an opportunity for palliative (symptom management) or supportive care concurrent with active treatment following diagnosis (i.e. not solely as a palliative or end-of-life measure) are considered eligible for this study.
* Does not have reliable access to Internet or sufficient personal data plan, and is not willing to participate in the Tablet Lending Program provided for this study.
* Does not have a working email address. BEHAVIORAL: Internet-based pain coping skills program
Cancer
Cancer pain, Survivor, Pain, Pain coping
Targeted Reversal of Inflammation in Pediatric Sepsis-induced MODS (TRIPS)
Mark Hall, MD - mark.hall@nationwidechildrens.org
NCT05267821
Inclusion Criteria:
* ≥ 40 weeks corrected gestational age to \< 18 years; AND
* Admission to the PICU or CICU; AND
* Onset of ≥ 2 new organ dysfunctions within the last 3 calendar days (compared to pre-sepsis baseline) as measured by the modified Proulx criteria; AND
* Documented or suspected infection as the MODS inciting event.
Exclusion Criteria:
* Weight \<3kg; OR
* Limitation of care order at the time of screening; OR
* Patients at high likelihood of progression to brain death in opinion of the clinical team; OR
* Moribund condition in which the patient is unlikely to survive the next 48 hours in opinion of the clinical team; OR
* Current or prior diagnosis of hemophagocytic lymphohistiocytosis or macrophage activation syndrome; OR
* Peripheral white blood cell count \< 1,000 cells/mm3 as the result of myeloablative therapy OR receipt of myeloablative therapy within the previous 14 days; OR
* Known allergy to anakinra, or E. coli-derived products; OR
* Known pregnancy; OR
* Lactating females; OR
* Receipt of anakinra within the previous 28 days; OR
* Resolution of MODS by MODS Day 2; OR
* Previous enrollment in the TRIPS study. DRUG: Anakinra, DRUG: Placebo
Pediatric Sepsis-induced Multiple Organ Dysfunction Syndrome (MODS)