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538 Study Matches

MT2015-29 : Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Adult Unrelated Donor for the Treatment of Hematological Disorders

The primary research element is to determine whether a graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide, tacrolimus and MMF will reduce the likelihood of chronic GVHD in patients receiving a standard hematopoietic myeloablative stem cell transplant. The treatment related components of this protocol are established clinical practices and are considered non-investigational. The primary endpoint is cumulative incidence of chronic GVHD requiring systemic immunosuppressive treatment at 1 year post-transplant.

Shernan Holtan
All
up to 60 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03314974
STUDY00001087
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Inclusion Criteria:

• Age: ≤ 60 years of age
• Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70
• Consent: Voluntary written consent (adult or legally authorized representative; or parental/guardian)
• Adequate Organ Function:
• Renal: Creatinine <2x upper limit of normal. Patients above this limit must have creatinine clearance ≥ 40 ml/min/1.73m2 as determined by an age-appropriate method, such as cystatin C GFR.
• Hepatic: Bilirubin, AST, alkaline phosphatase <4 times the upper limit of institutional normal
• Pulmonary: Diffusion capacity of oxygen, corrected for hemoglobin, > 50% of predicted. For pediatric patients not able to undergo PFTs or diffusion testing: O2 sat of >95% on room air
• Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 45%. For children not able to cooperate with MUGA or echocardiography, such should be clearly stated in the physician's documentation
• HIV Status: HIV infection with undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation Other
Inclusion Criteria:

• Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment.
• Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program
• Eligible Diseases and Status: Patients are eligible unless their treatment is to be guided by a higher priority protocol.
• Acute Leukemias: Must be in remission by morphology (≤5% blasts). Also a small percentage of blasts that is equivocal between marrow regeneration vs. early relapse are acceptable provided there are no associated cytogenetic markers consistent with relapse.
• Acute Myeloid Leukemia (AML) and related precursor neoplasms: 2nd or greater complete remission (CR); first complete remission (CR1) in patients > 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk.
• Favorable risk AML is defined as having one of the following:
• t(8,21) without cKIT mutation
• inv(16) or t(16;16) without cKIT mutation
• Normal karyotype with mutated NPM1 and wild type FLT-ITD
• Normal karyotype with double mutated CEBPA
• Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation
• Very high risk pediatric patients with AML: Patients <21 years, however, are eligible with (M2 marrow) with < 25% blasts in marrow after having failed one or more cycles of chemotherapy.
• Acute lymphoblastic leukemia (ALL)/lymphoma: second or greater CR; CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL.
• High risk ALL is defined as having one of the following:
• Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1
• 30 years of age or older at diagnosis
• White blood cell counts of greater than 30,000/mcL (B-ALL) or greater than 100,000/mcL (T-ALL) at diagnosis
• CNS leukemia involvement during the course of disease
• Slow cytologic response (>10% lymphoblasts in bone marrow on Day 14 of induction therapy)
• Evidence of persistent immonophenotypic or molecular minimal residual disease (MRD) at the end of induction and consolidation therapy
• Very high risk pediatric patients with ALL: patients <21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieve a complete remission.
• Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to one or more tyrosine kinase inhibitors.
• Plasma Cell Leukemia after initial therapy, in patients who have achieved at least a partial remission
• Myeloproliferative Neoplasms/Myelofibrosis, either primary as a result of polycythemia vera or essential thrombocythemia, with disease risk of intermediate or high-risk according to DIPSS criteria. Blasts must be <10% by bone marrow aspirate morphology.
• Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia, transfusion dependence, or high risk cytogenetics or molecular features. Blasts must be < 10% by a representative bone marrow aspirate morphology.
• Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting > 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for debulking chemotherapy before transplant.
• Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.
• Diffuse large Cell NHL > CR/> PR: Patients in CR/PR with initial short remission (<6 months) are eligible, or those who have failed/or are not eligible for autologous transplant.
• Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II < 1 year.
• Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin > 3 mg/L, may be considered for this protocol after initial therapy.
• Juvenile myelomonocytic leukemia
• Biphenotypic/Undifferentiated/Prolymphocytic Leukemias in first or subsequent CR.
• MRD positive leukemia (AML, ALL or accelerated/blast phase CML). Selected patients in morphologic CR, but with positive immunophenotypic (flow cytometry) or molecular evidence of MRD may be eligible if recent chemotherapy has not resulted in MRD negative status.
• Natural Killer Cell Malignancies
• Acquired Bone Marrow Failure Syndromes except for Fanconi Anemia or Dyskeratosis Congenita
• Other Leukemia Subtypes: A major effort in the field of hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
Exclusion Criteria:

• Chemotherapy refractory large cell and high grade NHL (i.e., progressive disease after > 2 salvage regimens)
• CML in blast crisis
• Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressing on salvage therapy.
• Evidence of progressive disease by imaging modalities or biopsy - persistent PET activity, though possibly related to lymphoma, is not an exclusion criterion in the absence of CT changes indicating progression.
• Active central nervous system malignancy
• if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
• Active HIV infection or known HIV positive serology
• active uncontrolled infection
• Pregnant or breastfeeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy.
Biological: HSCT with TBI Regimen, Biological: HSCT with Non-TBI Regimen
Acute Leukemia, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Lymphoma, Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myeloproliferative Neoplasms, Myelofibrosis, Myelodysplasia, Refractory Anemia, High Risk Anemia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Diffuse Large Cell Non Hodgkins Lymphoma, Lymphoblastic Lymphoma, Burkitt Lymphoma, High Grade Non-Hodgkin's Lymphoma, Adult, Multiple Myeloma, Juvenile Myelomonocytic Leukemia, Biphenotypic/Undifferentiated/Prolymphocytic Leukemias, MRD Positive Leukemia, Natural Killer Cell Malignancies, Acquired Bone Marrow Failure Syndromes
AML, ALL, MDS, NHL, CLL, CML, SLL, Clinics and Surgery Center (CSC)
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Neoadjuvant therapy for patients with high risk stage III melanoma: a pilot clinical trial (NeoACTIVATE)

To estimate the percentage of patients with stage III BRAFm melanoma that achieves a pathologic complete response after 12 weeks of neoadjuvant vemurafenib/cobimetinib/atezolizumab. To estimate the percentage of patients with stage III BRAFwt melanoma that achieves a pathologic complete response after 12 weeks of neoadjuvant cobimetinib/atezolizumab. Adjuvant phase primary objectives: To assess recurrence-free survival (RFS) in patients with stage III BRAFm melanoma after neoadjuvant vemurafenib/cobimetinib/atezolizumab, surgery, and adjuvant atezolizumab. To assess RFS in patients with stage III BRAFwt melanoma after neoadjuvant cobimetinib/atezolizumab, surgery, and adjuvant atezolizumab.

Evidio Domingo Musibay
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03554083
STUDY00004666
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Inclusion Criteria:

• PRE-REGISTRATION: High-risk stage III melanoma, defined as (any of the following):
• Recurrent nodal metastasis, or
• Clinically detectable nodal metastasis, or
• Metastatic involvement of more than one nodal basin
• NOTE: For the purpose of pre-registration, high-risk stage III melanoma is defined based on clinical and imaging assessment (positron emission tomography/computed tomography [PET/CT], CT, or magnetic resonance imaging [MRI]). Histologic confirmation of nodal metastatic disease is not needed at the time of pre-registration, provided there is histologic confirmation of primary melanoma or a prior lymph node metastasis.
• PRE-REGISTRATION: Willing to submit archival tissue from a lymph node biopsy or undergo a needle biopsy (with clip placement) for BRAF testing and for research purposes.
• PRE-REGISTRATION: Willing to forego anticancer treatments or investigational agents during pre-registration period.
• PRE-REGISTRATION: The following laboratory values obtained =< 28 days prior to pre-registration:
• Only for patients receiving therapeutic anticoagulation: stable anticoagulant regimen and stable international normalized ratio (INR).
• REGISTRATION: Histologic confirmation of stage III melanoma, as defined by the American Joint Committee on Cancer, 8th revised edition.
• REGISTRATION: Documentation of BRAFV600 mutation status in melanoma tumor tissue (archival or newly obtained) through use of a Clinical Laboratory Improvement Amendments (CLIA)-approved clinical mutation test.
• REGISTRATION: Surgically resectable disease, as determined by a melanoma surgical oncologist.
• REGISTRATION: Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
• REGISTRATION: Life expectancy >= 26 weeks.
• REGISTRATION: Absolute neutrophil count (ANC) >= 1500/mm^3 obtained =< 14 days prior to registration.
• REGISTRATION: Platelet count >= 100,000/mm^3 obtained =< 14 days prior to registration.
• REGISTRATION: Hemoglobin >= 9.0 g/dL obtained =< 14 days prior to registration.
• REGISTRATION: Direct bilirubin =< institutional upper limit of normal (ULN) obtained =< 14 days prior to registration.
• REGISTRATION: Aspartate transaminase (AST) and alanine transaminase (ALT) =< 2 x ULN obtained =< 14 days prior to registration.
• REGISTRATION: Alkaline phosphatase < 2.5 x ULN obtained =< 14 days prior to registration.
• REGISTRATION: Creatinine =< 1.5 x ULN or creatinine clearance (CrCl) >= 45 mL/min on the basis of measured CrCl from a 24-hour urine collection or Cockcroft-Gault glomerular filtration rate estimation obtained =< 14 days prior to registration.
• REGISTRATION: Arms A and B only: Left ventricular ejection fraction (LVEF) >= 50% or institutional lower limit of normal (LLN) =< 6 months prior to registration.
• REGISTRATION: Arms A and B only: Average corrected QT interval (QTc) =< 450 ms on triplicate 12 lead electrocardiography (ECG) =< 28 days prior to registration.
• NOTE: QTc intervals will be corrected using Fridericia's formula.
• REGISTRATION: Negative pregnancy test done =< 7 days prior to registration, for persons of childbearing potential only.
• REGISTRATION: For persons of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use a contraceptive method with a failure rate of < 1% per year during the treatment period and for 6 months after the last dose of study treatment.
• REGISTRATION: For persons able to father a child: agreement to remain abstinent (refrain from heterosexual intercourse with a person of childbearing potential) or use contraceptive measures, and agreement to refrain from donating sperm during the treatment period and for 6 months after the last dose of study treatment.
• REGISTRATION: Provide written informed consent.
• REGISTRATION: Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study).
• REGISTRATION: Willing to provide tissue, blood, and stool samples for correlative research purposes.
• REGISTRATION: Arm C Only: Negative serology for acute Epstein-Barr virus (EBV) infection (negative EBV viral capsid antigen [VCA] immunoglobulin M [IgM]).
Exclusion Criteria:

• PRE-REGISTRATION: Prior systemic anti-cancer therapy for melanoma (e.g., chemotherapy, hormonal therapy, targeted therapy, immunotherapy including anti-PD-1, anti-PDL1 agents, or other biologic therapies), with the following exceptions: adjuvant treatment with interferon, IL-2, granulocyte-macrophage colony-stimulating factor (GM-CSF) or vaccine therapies are allowed, if discontinued >= 28 days prior to pre-registration.
• PRE-REGISTRATION: Receiving any other investigational agent which would be considered as a treatment for the primary neoplasm.
• PRE-REGISTRATION: For patients with concurrent diagnosis of primary melanoma with nodal involvement, major surgical procedure other than lymph node biopsy or wide local excision of primary melanoma =< 4 weeks prior to pre-registration, or anticipation of need for a major surgical procedure for reasons other than melanoma during the course of the study.
• PRE-REGISTRATION: For patients with nodal recurrence, surgical procedure or anti-cancer therapy for this recurrence (other than lymph node biopsy) or anticipation of need for a major surgical procedure for reasons other than melanoma during the course of the study.
• PRE-REGISTRATION: Prior radiotherapy for melanoma.
• PRE-REGISTRATION: History of non-nodal melanoma metastasis or central nervous system (CNS) lesion(s) proven or clinically suspected to be metastasis.
• PRE-REGISTRATION: Active malignancy (other than melanoma) or malignancy =< 3 years prior to pre-registration.
• NOTE: Exceptions: Asymptomatic papillary thyroid cancer (not requiring treatment), resected basal cell carcinoma (BCC), resected cutaneous squamous cell carcinoma (SCC), resected carcinoma in situ of the cervix, resected carcinoma in situ of the breast, in situ prostate cancer, non-muscle-invasive bladder cancer, Stage I uterine cancer, or other curatively treated malignancies from which the patient has been disease-free for at least 3 years prior to pre registration.
• PRE-REGISTRATION: Prior allogeneic stem cell or solid organ transplantation.
• PRE-REGISTRATION: History of idiopathic pulmonary fibrosis, organizing pneumonia (e.g., bronchiolitis obliterans), drug-induced pneumonitis, or idiopathic pneumonitis, or evidence of active pneumonitis on screening chest CT scan.
• PRE-REGISTRATION: History of autoimmune disease requiring systemic immunosuppressive or immune-modulatory therapy =< 5 years prior to pre-registration.
• NOTE: Exceptions are allowed for hypothyroidism on thyroid replacement therapy; or Type 1 diabetes on insulin regimen.
• PRE-REGISTRATION: Active psoriasis requiring therapy (systemic or topical).
• PRE-REGISTRATION: Known clinically significant liver disease, including alcoholism, cirrhosis, fatty liver, and other inherited liver disease as well as active viral disease.
• PRE-REGISTRATION: Arms A and B only: History of or evidence of retinal pathology on ophthalmologic examination including but not limited to:
• Neurosensory retinal detachment
• Central serous chorioretinopathy
• Retinal vein occlusion (RVO)
• Neovascular macular degeneration
• PRE-REGISTRATION: Immunocompromised patients and patients known to be human immunodeficiency virus (HIV) positive and currently receiving antiretroviral therapy.
• NOTE: Patients known to be HIV positive, but without clinical evidence of an immunocompromised state, are eligible for this trial.
• PRE-REGISTRATION: Uncontrolled intercurrent illness including, but not limited to:
• Ongoing or active infection (including but not limited to tuberculosis)
• Clinically significant cardiac dysfunction including:
• Symptomatic congestive heart failure defined as New York Heart Association class II or higher
• Unstable angina pectoris or new-onset angina =< 3 months prior to pre-registration
• Unstable cardiac arrhythmia
• Myocardial infarction =< 3 months prior to pre-registration
• Congenital long QT syndrome
• Clinically significant stroke, reversible ischemic neurological defect, or transient ischemic attack =< 6 months prior to pre-registration
• Any grade 3 hemorrhage or bleeding event =< 4 weeks prior to pre-registration
• Uncontrolled diabetes or symptomatic hyperglycemia
• Psychiatric illness/social situations that, in the judgement of the investigator, would: a) limit compliance with study requirements, or b) make the patient inappropriate for entry into this study, or c) interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens.
• PRE-REGISTRATION: Known hypersensitivity to biopharmaceutical agents produced in Chinese hamster ovary cells (example [ex]: recombinant follicle-stimulating hormone [FSH]).
• PRE-REGISTRATION: Known hypersensitivity to any components of the atezolizumab (all arms), tiragolumab (Arm C only), cobimetinib (Arms A and B only), or vemurafenib (Arms A and B only) formulations.
• PRE-REGISTRATION: History of severe allergic, anaphylactic or other hypersensitivity reactions to chimeric or humanized antibodies or fusion proteins.
• REGISTRATION: Received anticancer treatments or investigational agents during pre-registration period.
• REGISTRATION: Clinically suspected non-nodal metastatic melanoma.
• REGISTRATION: Arm A only: For BRAF-mutant patients only: anticipated use of any concomitant medication =< 7 days prior to registration that is known to cause QT prolongation (which may lead to torsade de pointes).
• REGISTRATION: Arms A and B only: History of malabsorption or other clinically significant metabolic dysfunction that may interfere with absorption of oral study treatment or inability or unwillingness to swallow oral medication.
• REGISTRATION: Signs or symptoms of infection or has received antibiotics =< 14 days prior to registration.
• NOTE: Patients receiving prophylactic antibiotics (e.g., to prevent a urinary tract infection or chronic obstructive pulmonary disease exacerbation) are eligible for the study.
• REGISTRATION: Any of the following because this study involves investigational agents whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown:
• Pregnant persons
• Nursing persons
• Persons of childbearing potential who are unwilling to employ adequate contraception
• REGISTRATION: Treatment with a live, attenuated vaccine =< 4 weeks prior to registration, or anticipation of need for such a vaccine during the course of the study.
• REGISTRATION: Treatment with systemic immunosuppressive medication (including, but not limited to, prednisone, cyclophosphamide, azathioprine, methotrexate, thalidomide, and anti-tumor necrosis factor (TNF)-alpha agents) =< 2 weeks prior to registration, or anticipation of need for systemic immunosuppressive medication during the course of the study.
• NOTE: Patients who have received acute, low-dose systemic steroids (=< 10 mg/day oral prednisone or equivalent) prior to registration or a one-time pulse dose of systemic immunosuppressant medication (e.g., 48 hours of corticosteroids for a contrast allergy) are eligible for the study.
• NOTE: The use of inhaled corticosteroids for chronic obstructive pulmonary disease or asthma, mineralocorticoids (e.g., fludrocortisone), or low-dose corticosteroids for patients with orthostatic hypotension or adrenocortical insufficiency is allowed.
• REGISTRATION: Requirement for concomitant therapy or food that is prohibited during the study.
• REGISTRATION: Arms A and B only: Inability to abstain from alcohol during neoadjuvant phase.
• REGISTRATION: Arm C only: Known Epstein-Barr virus (EBV) infection.
• NOTE: Patients with symptoms such as splenomegaly, fever, sore throat, non-malignant cervical lymphadenopathy, and/or tonsillar exudate, should undergo an EBV polymerase chain reaction (PCR) test to screen for acute infection or suspected chronic active infection. Patients with a positive EBV PCR test are excluded.
Drug: Atezolizumab, Drug: Cobimetinib, Biological: Tiragolumab, Drug: Vemurafenib
Clinical Stage III Cutaneous Melanoma AJCC v8, Pathologic Stage III Cutaneous Melanoma AJCC v8, Pathologic Stage IIIA Cutaneous Melanoma AJCC v8, Pathologic Stage IIIB Cutaneous Melanoma AJCC v8, Pathologic Stage IIIC Cutaneous Melanoma AJCC v8, Pathologic Stage IIID Cutaneous Melanoma AJCC v8
Clinics and Surgery Center (CSC)
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Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options (STARTRK-NG)

This is a Phase 1/2 multicenter, open-label dose escalation study in pediatrics patients (<18 years) with relapsed or refractory extracranial solid tumors (Phase 1; Part A), with additional expansion cohorts (Phase 2) in patients with primary brain tumors harboring NTRK1/2/3 or ROS1 gene fusions (Part B), and extracranial solid tumors harboring NTRK1/2/3 or ROS1 gene fusions (Part D).

Emily Greengard
All
up to 18 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT02650401
STUDY00001185
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Inclusion Criteria:

• Disease status:
• Phase 1 portion (closed): Participants must have measurable or evaluable disease, as defined by RECIST v1.1
• Phase 2 portion:
• Part B: Participants must have measurable or evaluable disease, as defined by RANO
• Part C (closed): Participants must have measurable or evaluable disease, as defined by RECIST v1.1 ± Curie Scale
• Part D: Participants must have measurable or evaluable disease, as defined by RECIST v1.1
• Part E (closed): Participants must have measurable or evaluable disease, as defined by RECIST v1.1 ± Curie Scale or RANO
• Tumor type:
• Phase 1 portion: * Part A: Relapsed or refractory extracranial solid tumors
• Phase 2 portion
• Part B: Primary brain tumors with NTRK1/2/3 or ROS1 gene fusions; gene fusions are defined as those predicted to translate into a fusion protein with a functional TRKA/B/C or ROS1 kinase domain, without a concomitant second oncodriver as determined by a nucleic acid-based diagnostic testing method
• Part D: Extracranial solid tumors (including NB) with NTRK1/2/3 or ROS1 gene fusions; gene fusions are defined as those predicted to translate into a fusion protein with a functional TRKA/B/C or ROS1 kinase domain, without a concomitant second oncodriver as determined by a nucleic acid-based diagnostic testing method
• Histologic/molecular diagnosis of malignancy at diagnosis or the time of relapse
• Archival tumor tissue from diagnosis or, preferably, at relapse
• Performance status: Lansky or Karnofsky score ≥ 60% and minimum life expectancy of at least 4 weeks
• Prior therapy: Participants must have a disease that is locally advanced, metastatic, or where surgical resection is likely to result in severe morbidity, and who have no satisfactory treatment options for solid tumors and primary CNS tumors that are neurotrophic tyrosine receptor kinase (NTRK) or ROS1 fusion-positive
• Participants must have recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to enrollment
• Adequate organ and neurologic function
• Females of childbearing potential must have a negative serum pregnancy test during screening and be neither breastfeeding nor intending to become pregnant during study participation. Agreement to remain abstinent or use use combined contraceptive methods prior to study entry, for the duration of study participation and in the following 90 days after discontinuation of study treatment.
• For male participants with a female partner of childbearing potential or a pregnant female partner: Agreement to remain abstinent or use a condom during the treatment period and for at least 3 months after the last dose of study drug
Exclusion Criteria:

• Receiving other experimental therapy
• Known congenital long QT syndrome
• History of recent (3 months) symptomatic congestive heart failure or ejection fraction ≤50% at screening
• Known active infections
• Familial or personal history of congenital bone disorders, bone metabolism alterations or osteopenia
• Receiving Enzyme Inducing Antiepileptic Drugs (EIAEDs) within 14 days of first dose.
• Prior treatment with approved or investigational TRK or ROS1 inhibitors
• Known hypersensitivity to entrectinib or any of the other excipients of the investigational medicinal product
• Patients with NB with bone marrow space-only disease
• Incomplete recovery from acute effects of any surgery prior to treatment.
• Active gastrointestinal disease or other malabsorption syndromes that would impact drug absorption.
• Other severe acute or chronic medical or psychiatric condition or lab abnormality that may increase the risk associated with study participation, drug administration or may interfere with the interpretation of study results.
Drug: Entrectinib
Solid Tumors, CNS Tumors
TRK, Tyrosine kinase, NTRK, NTRK1, NTRK2, NTRK3, ROS1, ALK, Pediatric, Relapsed, Refractory, Solid Tumor, Metastatic Cancer, Gene rearrangement, Neuroblastoma, Infantile fibrosarcoma, Secretory breast cancer, Congenital mesoblastic nephroma, Pontine glioma, Brain tumors, CNS tumors, Sarcoma, Ewing sarcoma, Glial tumors, Salivary Gland Cancer (MASC), Papillary thyroid cancer, Medulloblastoma, Wilms tumor (anaplastic)
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RANDOMIZED CONTROLLED TRIAL COMPARING RELAPSE RATES BETWEEN STANDARD SYMPTOMATIC URINARY STONE REMOVAL AND STANDARD REMOVAL WITH ADDITIONAL URETERORENOSCOPIC CLEARING OF NON-SYMPTOMATIC STONES IN THE KIDNEY

Michael Borofsky
All
21 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02210650
1610M98561
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Inclusion Criteria:

• Scheduled to undergo surgery (URS or PCNL) for a primary stone.
• Computed tomography (CT) exam within the 90-day pre-operative period
• Small (≤ 6mm) asymptomatic stones in visible on KUB or CT (i.e., calcium stones) in the contralateral kidney for a primary renal stone or ipsilateral kidney for primary ureteral stone.
• Recurrent (having had previous stones) or multiple (simultaneous bilateral stones) stones
• Able to give informed consent
• Age 21 years or older
Exclusion Criteria:

• Inability to give informed consent
• Age less than 21 years
• Stones not visible on KUB or CT
• Patients with systemic disease or renal anatomical disorders (RTA, primary hyperparathyroidism, sarcoidosis, enteric hyperoxaluria, medullary sponge kidney)
• Any condition (eg, psychiatric illness) or situation that, in the investigator's opinion, could put the
• subject at significant risk, confound the study results, or interfere significantly with the subject's
• participation in the study.
• Unwilling to participate.
Procedure: Symptomatic stone removal, Procedure: Asymptomatic kidney stones and ureteral stone removed
Ureteral Stones, Kidney Stones
kidney stone
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Dysphonia, distress, and perceived control: Technology-based assessment and intervention

This is a randomized controlled trial that seeks to pilot test, in patients with voice disorders (dysphonia), an existing online intervention that reduces the effects of stress by increasing perceived control. We propose to examine the acceptability and usability of our adapted intervention- Vocal Outcome Improvement through Control Enhancement (VOICE) to see if the adapted intervention is successful at: a) increasing perceived control over voice-related stressors b) decreasing stress and distress resulting from voice problems. Our goal is to help participants who have a voice problem achieve better voice and quality of life outcomes.

Stephanie Misono
All
18 Years to 80 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03576365
1507S75003
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Inclusion Criteria:

• Patients who are seen at the Otolaryngology Clinic (University of Minnesota Health Clinics and Surgery Center, or Fairview Hospital Maple Grove or Southdale) with Muscle Tension Dysphonia
• Patients between 18-80 years of age experiencing a voice problem
• Scored high (>10) on the Voice Handicap Index (VHI-10)
• Has the ability to complete informed consent process
• Interested in using an online program/intervention
• Has reliable access to internet
Exclusion Criteria:

• Patients who have clinical need for concurrent treatment will be excluded to decrease confounding introduced by having multiple simultaneous interventions. This may include:
• Concurrent laryngeal lesion requiring immediate operative or other intervention
• Concurrent participation in speech therapy
• Concurrent need for new medications that may directly affect voice-related symptoms
• Participant has a Speech Language Pathology visit scheduled in two weeks or less from when they will start the program (if applicable)
• Unable to read English (as determined by their ability to complete the clinic intake forms)
• Female and pregnant
• Prisoner
• Unable to provide informed consent (e.g., patients with dementia)
Behavioral: VOICE Intervention Arm, Behavioral: Information-Only Arm
Functional Dysphonia, Voice Disorders, Dysphonia
Perceived Control, Distress
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Spinal Manipulation and Patient Self-Management for Preventing Acute to Chronic Back Pain (PACBACK) (PACBACK)

The long-term objective is to reduce overall low back pain (LBP) burden by testing scalable, first-line, non-pharmacologic strategies that address the biopsychosocial aspects of acute/sub-acute LBP and prevent transition to chronic LBP. This is a novel randomized hybrid trial addressing both effectiveness and implementation. A total of 1180 patients will be enrolled with nonspecific LBP of 2-12 weeks duration, at medium or high risk of developing chronic LBP. This multi-site, predominantly pragmatic, phase III trial has two main aims. Aim 1 will assess the effectiveness of Spinal Manipulation Therapy (SMT), Supported Self-Management (SSM), and SMT+SSM relative to Medical Care (MC) in a randomized trial using a 2x2 factorial design. Aim 2 will use mixed methods to gather data about influences on the interventions that could affect interpretation of results and future implementation.

Gert Bronfort
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03581123
SITE00000150
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Inclusion Criteria:

• At least 18 years of age
• Acute or sub-acute low back pain
• Average low back pain severity ≥3 on the 0-10 numerical rating scale over 7 days
• Medium or high risk for persistent disabling back pain according to the STarT Back screening tool
• Ability to read and write fluently in English
Exclusion Criteria:

• Non-mechanical causes of low back pain
• Contraindications to study treatments (e.g,. surgical fusion of lumbar spine)
• Active management of current episode of low back pain by another healthcare provider
• Serious co-morbid health condition that either requires medical attention or has a risk for general health decline over the next year
• Pregnancy, current or planned during study period and nursing mothers
• Inability or unwillingness to give written informed consent
Behavioral: Supported-Self Management (SSM), Other: Spinal Manipulation Therapy (SMT), Combination Product: SMT + SSM, Drug: Standard Medical Care (SMC)
Acute Pain, Low Back Pain, Mechanical
acute/subacute low back pain, randomized clinical trial, self-management, behavioral modification, spinal manipulation therapy, standard medical care, secondary prevention
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Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

This phase II trial studies how well trametinib works in treating patients (≥ 2 years and < 22 years of age) with juvenile myelomonocytic leukemia that has come back or does not respond to treatment. Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Emily Greengard
All
1 Month to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03190915
STUDY00003883
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Inclusion Criteria:

• Patients must have had histologic verification of juvenile myelomonocytic leukemia (JMML) at original diagnosis and currently have relapsed or refractory disease; the diagnosis is made based on the following criteria
• JMML category 1 (all of the following): the diagnostic criteria must include all features in category 1 and EITHER (i) one of the features in category 2 OR (ii) two features from category 3 to make the diagnosis
• Splenomegaly
• > 1000 (1 x 10^9/uL) circulating monocytes
• < 20% blasts in the bone marrow or peripheral blood
• Absence of the t(9;22) or BCR/ABL fusion gene
• JMML category 2 (at least one of the following if at least two category 3 criteria are not present):
• Somatic mutation in RAS or PTPN11
• Clinical diagnosis of NF1 or NF1 gene mutation
• Homozygous mutation in CBL
• Monosomy 7
• JMML category 3 (at least two of the following if no category 2 criteria are met):
• Circulating myeloid precursors
• White blood cell count, > 10 000 (10 x 10^9/ uL)
• Increased hemoglobin F for age
• Clonal cytogenetic abnormality
• GM-CSF hypersensitivity
• Patients with refractory or relapsed JMML must have had at least one cycle of intensive frontline therapy or at least 2 cycles of a deoxyribonucleic acid (DNA) demethylating agent with persistence of disease, defined by clinical symptoms or the presence of a clonal abnormality; frontline therapy is defined as one cycle of intravenous chemotherapy that includes of any of the following agents: fludarabine, cytarabine, or any anthracycline but specifically excludes oral 6-mercaptopurine; frontline therapy will also include any conditioning regimen as part of a stem cell transplant; patients who transform to AML at any point with more than 20% blasts are not eligible for this trial
• Patients must have a Lansky or Karnofsky performance status score of >= 50, corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study enrollment
• Myelosuppressive chemotherapy: patients must have completely recovered from all acute toxic effects of chemotherapy, immunotherapy or radiotherapy prior to study enrollment; at least 14 days must have elapsed since the completion of cytotoxic therapy, with the exception of hydroxyurea
• Note: cytoreduction with hydroxyurea can be initiated and continued for up to 24 hours prior to the start of protocol therapy
• Hematopoietic growth factors: at least 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or 7 days for short-acting growth factor; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
• Biologic (anti-neoplastic agent): at least 7 days must have elapsed since completion of therapy with a biologic agent; for agents that have known adverse events occurring beyond 7 days after administration, this period prior to enrollment must be extended beyond the time during which adverse events are known to occur
• Monoclonal antibodies:
• At least 30 days after the completion of any type of immunotherapy, e.g. tumor vaccines
• At least 3 half-lives must have elapsed since prior therapy that included a monoclonal antibody
• Radiotherapy:
• >= 2 weeks must have elapsed since local palliative external radiation therapy (XRT) (small port)
• >= 6 months must have elapsed if prior craniospinal XRT was received, if >= 50% of the pelvis was irradiated, or if traumatic brain injury (TBI) was received
• >= 4 weeks must have elapsed if other substantial bone marrow irradiation was given
• Stem cell transplant or rescue without TBI: no evidence of active graft versus (vs.) host disease and >= 3 months must have elapsed since transplant; >= 4 weeks must have elapsed since any donor lymphocyte infusion
• Patients must not be known to be refractory to red blood cell or platelet transfusions
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• Age: Maximum serum creatinine (mg/dL)
• 1 month to < 6 months: 0.4 (male) 0.4 (female)
• 6 months to < 1 year: 0.5 (male) 0.5 (female)
• 1 to < 2 years: 0.6 (male) 0.6 (female)
• 2 to < 6 years: 0.8 (male) 0.8 (female)
• 6 to < 10 years: 1 (male) 1 (female)
• 10 to < 13 years: 1.2 (male) 1.2 (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
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 3 x ULN (=< 135 U/L) (for the purpose of this study, the ULN for SGPT is 45 U/L)
• Serum albumin >= 2 g/dL
• Shortening fraction of >= 27% by echocardiogram OR ejection fraction of >= 50% by multi-gated acquisition (MUGA)
• Corrected QT (by Bazett's formula [QTcB]) interval < 450 msecs
• Patients must be able to swallow tablets or liquid; use of a nasogastric or gastrostomy (G) tube is also allowed
Exclusion Criteria:

• Patients who are pregnant or breast-feeding are not eligible for this study as there is yet no available information regarding human fetal or teratogenic toxicities; negative pregnancy tests must be obtained in girls who are post-menarchal; patients of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for the duration of study therapy; women of childbearing potential should be advised to use effective contraception for 4 months after the last dose of trametinib; trametinib may also potentially be secreted in milk and therefore breastfeeding women are excluded; female patients should not breastfeed during treatment with trametinib, and for 4 months following the last dose; male patients must use a condom during intercourse and agree not to father a child during therapy and for 4 months following discontinuation of trametinib to avoid unnecessary exposure of trametinib to the fetus
• Concomitant Medications
• Corticosteroids: patients requiring corticosteroids who have not been on a stable or decreasing dose of corticosteroid for the 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Note: hydrocortisone used as a pre-medication to prevent transfusion related reactions is not considered a concomitant corticosteroid
• Investigational drugs: patients who are currently receiving another investigational drug are not eligible
• Anti-cancer agents: patients who are currently receiving other anti-cancer agents are not eligible (except patients receiving hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy)
• Anti-graft versus host disease (GVHD) or agents to prevent organ rejection post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial
• Cardiac medications: any medications for treatment of left ventricular systolic dysfunction
• Patients who have an uncontrolled infection are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
• Patients with a history of hepatic sinusoid obstructive syndrome (veno-occlusive disease) within the prior 3 months are not eligible
• Patients with a history of or current evidence/risk of retinal vein occlusion (RVO) or central serous retinopathy (CSR) are not eligible
• Patients with a history of RVO or CSR, or predisposing factors to RVO or CSR (e.g., uncontrolled glaucoma or ocular hypertension
• Patients with uncontrolled systemic disease(s) such as hypertension or diabetes mellitus are not eligible; blood pressure must be =< the 95th percentile for age, height, and gender
• Patients with a history of allergic reaction attributed to compounds of similar chemical or biologic composition to the MEK inhibitor, trametinib are not eligible
• Patients with a clinical diagnosis of Noonan syndrome are not eligible; Note: patients with Casitas B-lineage lymphoma (CBL) syndrome, also known as Noonan-like syndrome, are eligible to enroll
Drug: Trametinib
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
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An Open-Label, Single-Arm, Multicenter Study of Intracerebral Administration of Adeno-Associated Viral Vectors Serotype rh10 Carrying the Human N sulfoglucosamine sulfohydrolase (SGSH) cDNA for the Treatment of Mucopolysaccharidosis Type IIIA (AAVance)

Chester Whitley, MD, PhD
All
6 Months and over
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT03612869
STUDY00004084
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Inclusion Criteria:

• Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene mutations
• Cognitive DQ score on BSID-III: 50% and above
Exclusion Criteria:

• Homozygous for the S298P mutation or non-classical severe form of MPS IIIA, based on investigator's judgement.
• Participation in another gene or cell therapy clinical trial.
• Past use of SGSH enzyme replacement therapy for a period exceeding 3 months. A washout period of at least 2 months is required prior to screening.
• Current participation in a clinical trial of another investigational medicinal product.
• History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following surgery.
• Any condition that would contraindicate treatment with immunosuppressants such as tacrolimus, mycophenolate mofetil or steroids.
Drug: LYS-SAF302
Mucopolysaccharidosis Type IIIA
MPS IIIA, Sanfilippo syndrome Type A, Mucopolysaccharidosis Type IIIA, Lysosomal Storage Disease
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MT-2018-20: COG AALL1631 - International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones

This randomized phase III trial studies how well imatinib mesylate and combination chemotherapy work in treating patients (> 1 year and < 21 years) with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia. Imatinib mesylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving imatinib mesylate and combination chemotherapy may work better in treating patients with Philadelphia chromosome positive acute lymphoblastic leukemia.

Peter Gordon
All
1 Year to 21 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03007147
STUDY00003635
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Inclusion Criteria:

• Diagnostic samples will be collected and analyzed according to the procedures of the National front-line protocol
• Patients should be enrolled on National ALL protocol prior to enrollment on EsPhALL2017/COGAALL1631. Regardless of initial front-line protocol baseline diagnostic samples must be available to develop an MRD probe
• BCR-ABL1 fusion (Ph+): newly diagnosed ALL (B-ALL or T-ALL) or mixed phenotypic acute leukemia (MPAL meeting 2016 World Health Organization [WHO] definition) with definitive evidence of BCR-ABL1 fusion by karyotype, fluorescence in situ hybridization (FISH) and/or reverse transcriptase (RT)-PCR
• ABL-class fusion: newly diagnosed B-ALL with definitive evidence of ABL-class fusions identified according to National/Center procedures of each participating country. ABL-class fusions are defined as those involving the following genes: ABL1, ABL2, CSF1R, PDGFRB, PDGFRA, LYN. Methods of detection include fluorescence in-situ hybridization (FISH, e.g. using break-apart or colocalization signals probes), multiplex or singleplex reverse-transcription polymerase chain reaction (RT-PCR), whole transcriptome or panel-based ribonucleic acid (RNA)-sequencing
• Regardless of initial front-line protocol, laboratory reports detailing evidence of BCR-ABL1 or ABL-class fusion must be available for the National Trial Unit
• Ph+ ALL patients must have previously started induction therapy, which includes vincristine, a corticosteroid, usually PEG-L-asparaginase, with or without anthracycline, and/or other standard cytotoxic chemotherapy
• Ph+ ALL patients have not received more than 14 days of multiagent induction therapy beginning with the first dose of vincristine
• Ph+ ALL patients may have started imatinib prior to study entry but have not received more than 14 days of imatinib
• ABL-class fusion patients must have previously completed the 4 or 5 weeks of multiagent Induction chemotherapy
• ABL-class fusion patients may have started imatinib during Induction IA, at the same time of or after the first vincristine dose
• Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2
• Direct bilirubin =< 2.0 mg/dL
• Shortening fraction of >= 27% by echocardiogram
• Ejection fraction of >= 50% by radionuclide angiogram or echocardiogram
• Corrected QT interval, QTc < 480 msec
• Note: Repeat echocardiogram and electrocardiogram are not required if they were performed at or after initial ALL diagnosis, before study enrollment
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or serum creatinine within normal limits based on age/gender, as follows:
• 1 to < 2 years: maximum serum creatinine 0.6 mg/dL (both male and female)
• 2 to < 6 years: maximum serum creatinine 0.8 mg/dL (both male and female)
• 6 to < 10 years: maximum serum creatinine 1 mg/dL (both male and female)
• 10 to < 13 years: maximum serum creatinine 1.2 mg/dL (both male and female)
• 13 to < 16 years: maximum serum creatinine 1.5 mg/dL (male), 1.4 mg/dL (female)
• >= 16 years: maximum serum creatinine 1.7 mg/dL (male), 1.4 mg/dL (female)
Exclusion Criteria:

• Known history of chronic myelogenous leukemia (CML)
• ALL developing after a previous cancer treated with cytotoxic chemotherapy
• Active, uncontrolled infection, or active systemic illness that requires ongoing vasopressor support or mechanical ventilation
• Down syndrome
• Pregnancy and breast feeding
• 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 treatment according to protocol
• Patients with congenital long QT syndrome, history of ventricular arrhythmias or heart block
• Prior treatment with dasatinib, or any TKI inhibitor other than imatinib
• All patients and/or their parents or legal guardians must sign a written informed consent
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation, Drug: Cyclophosphamide, Drug: Cytarabine, Drug: Daunorubicin Hydrochloride, Drug: Dexamethasone, Drug: Dexrazoxane Hydrochloride, Drug: Doxorubicin, Drug: Etoposide, Biological: Filgrastim, Drug: Ifosfamide, Drug: Imatinib Mesylate, Other: Laboratory Biomarker Analysis, Drug: Leucovorin Calcium, Drug: Mercaptopurine, Drug: Mercaptopurine, Drug: Methotrexate, Drug: Methylprednisolone, Drug: Pegaspargase, Drug: Prednisolone, Other: Questionnaire Administration, Drug: Therapeutic Hydrocortisone, Drug: Thioguanine, Drug: Vincristine Sulfate
Acute Lymphoblastic Leukemia, B Acute Lymphoblastic Leukemia, Mixed Phenotype Acute Leukemia, T Acute Lymphoblastic Leukemia
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COG AHEP1531 - Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)

This partially randomized phase II/III trial studies how well cisplatin and combination chemotherapy works in treating children and young adults (≤ 30 years of age) with hepatoblastoma or liver cancer after surgery. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan, sorafenib, gemcitabine and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving combination chemotherapy after surgery may kill more tumor cells.

Emily Greengard
All
up to 30 Years old
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT03533582
STUDY00003718
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Inclusion Criteria:

• Patients in Group F must have a body surface area (BSA) >= 0.6 m^2
• 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 who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must be newly diagnosed with histologically-proven primary pediatric hepatic malignancies including hepatoblastoma or hepatocellular carcinoma, except as noted below; patients with a diagnosis of hepatocellular neoplasm, not otherwise specified, should be classified and treated per hepatoblastoma treatment arms; note that rapid central pathology review is required in some cases; please note: all patients with histology as assessed by the institutional pathologist consistent with pure small cell undifferentiated (SCU) HB will be required to have testing for INI1/SMARCB1 by immunohistochemistry (IHC) according to the practices at the institution
• Patients with histology consistent with pure SCU must have positive INI1/SMARCB1 staining
• For all Group A patients, WDF status as determined by rapid review will be used to further stratify patients to Group A1 or A2
• For Groups B, C and D, rapid review is required if patients are either >= 8 years of age or have an alphafetoprotein (AFP) =< 100 at diagnosis
• For all Groups E and F patients, rapid central pathology review is required
• In emergency situations when a patient meets all other eligibility criteria and has had baseline required observations, but is too ill to undergo a biopsy safely, the patient may be enrolled without a biopsy
• Clinical situations in which emergent treatment may be indicated include, but are not limited to, the following circumstances:
• Anatomic or mechanical compromise of critical organ function by tumor (e.g., respiratory distress/failure, abdominal compartment syndrome, urinary obstruction, etc.)
• Uncorrectable coagulopathy
• For a patient to maintain eligibility for AHEP1531 when emergent treatment is given, the following must occur:
• The patient must have a clinical diagnosis of hepatoblastoma, including an elevated alphafetoprotein (AFP), and must meet all AHEP1531 eligibility criteria at the time of emergent treatment
• Patient must be enrolled on AHEP1531 prior to initiating protocol therapy; a patient will be ineligible if any chemotherapy is administered prior to AHEP1531 enrollment
• Note: If the patient receives AHEP1531 chemotherapy emergently PRIOR to undergoing a diagnostic biopsy, pathologic review of material obtained in the future during either biopsy or surgical resection must either confirm the diagnosis of hepatoblastoma or not reveal another pathological diagnosis to be included in the analysis of the study aims
• Patients may have had surgical resection of the hepatic malignancy prior to enrollment; all other anti-cancer therapy for the current liver lesion is prohibited
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 60 mL/min/1.73 m^2 or
• A serum creatinine based on age/gender as follows:
• 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: 06 (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 =< 5 x upper limit of normal (ULN) for age
• Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) < 10 x upper limit of normal (ULN) for age
• Shortening fraction of >= 28% by echocardiogram (for patients on doxorubicin-containing regimens [Groups C, D, E2, and F] assessed within 8 weeks prior to study enrollment) or
• Ejection fraction of >= 47% by echocardiogram or radionuclide angiogram (for patients on doxorubicin-containing regimens [Groups C, D, E2, and F] assessed within 8 weeks prior to study enrollment)
• Group F patients only: QT/corrected QT (QTc) interval =< 450 milliseconds for males and =< 470 milliseconds for females (assessed within 8 weeks prior to study enrollment)
• Normal pulmonary function tests (including diffusion capacity of the lung for carbon monoxide [DLCO]) if there is clinical indication for determination (e.g. dyspnea at rest, known requirement for supplemental oxygen) (for patients receiving chemotherapy [Groups A, B, C, D, E2, F]); for patients who do not have respiratory symptoms or requirement for supplemental oxygen, pulmonary function tests (PFTs) are NOT required
• 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:

• Prior chemotherapy or tumor directed therapy (i.e. radiation therapy, biologic agents, local therapy (embolization, radiofrequency ablation, and laser); therefore, patients with a pre-disposition syndrome who have a prior malignancy are not eligible
• Patients who are currently receiving another investigational drug
• Patients who are currently receiving other anticancer agents
• Patients with uncontrolled infection
• Patients who previously received a solid organ transplant, other than those who previously received an orthotopic liver transplantation (OLT) as primary treatment of their hepatocellular carcinoma
• Patients with hypersensitivity to any drugs on their expected treatment arm
• Group C: Patients who have known deficiency of dihydropyrimidine dehydrogenase (DPD)
• Group D:
• Patients with chronic inflammatory bowel disease and/or bowel obstruction
• Patients with concomitant use of St. John's wort, which cannot be stopped prior to the start of trial treatment
• Group F:
• Patients with peripheral sensitive neuropathy with functional impairment
• Patients with a personal or family history of congenital long QT syndrome
• These criteria apply ONLY to patients who may receive chemotherapy (all groups other than Group E1):
• 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
• Note for Group F: patients of childbearing potential should use effective birth control during treatment with sorafenib and for at least 2 weeks after stopping treatment
Drug: Carboplatin, Drug: Cisplatin, Drug: Doxorubicin, Drug: Etoposide, Drug: Fluorouracil, Drug: Gemcitabine, Drug: Irinotecan, Other: Laboratory Biomarker Analysis, Drug: Oxaliplatin, Other: Patient Observation, Drug: Sorafenib, Drug: Vincristine Sulfate
Childhood Hepatocellular Carcinoma, Childhood Malignant Liver Neoplasm, Fibrolamellar Carcinoma, Hepatoblastoma, Hepatocellular Malignant Neoplasm, Not Otherwise Specified
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COG ACNS1422 - A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients

Patients greater than or equal to 3 years of age and < 22 years of age with newly diagnosed type of brain tumor called WNT)/Wingless (WNT)-driven medulloblastoma using reduced craniospinal radiotherapy.

Christopher Moertel, MD
All
3 Years to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02724579
STUDY00002501
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Inclusion Criteria:

• Patients must be newly diagnosed and have:
• Eligibility confirmed by rapid central pathology and molecular screening review on APEC14B1:
• Classical histologic type (non LC/A) WNT medulloblastoma
• Positive nuclear beta-catenin by immunohistochemistry (IHC)
• Positive for CTNNB1 mutation
• Negative for MYC and MYCN by fluorescence in situ hybridization (FISH)
• Patient must have negative lumbar cerebrospinal fluid (CSF) cytology
• Note: CSF cytology for staging should be performed no sooner than 14 days post operatively to avoid false positive CSF; ideally, CSF should be obtained between day 14 and day 21 to allow for final staging status before enrollment onto the study; patients with positive CSF cytology obtained 0 to 14 days after surgery should have cytology repeated to determine eligibility and final CSF status; patients with negative CSF cytology from lumbar puncture obtained 0 to 14 days after surgery do not need cytology repeated; patients with negative CSF cytology from lumbar puncture obtained prior to surgery do not need cytology repeated post-operatively
• Patients must have eligibility confirmed by Rapid Central Imaging Review on APEC14B1; patients must have =< 1.5 cm^2 maximal cross-sectional area of residual tumor; whole brain magnetic resonance imaging (MRI) with and without gadolinium and spine MRI with gadolinium must be performed
• Patients must be enrolled, and protocol therapy must be projected to begin, no later than 36 days after definitive diagnostic surgery (day 0)
• Peripheral absolute neutrophil count (ANC) >= 1000/uL
• Platelet count >= 100,000/uL (transfusion independent)
• Hemoglobin >= 10.0 g/dL (may receive red blood cell [RBC] transfusions)
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• 3 to < 6 years of age: maximum (max) serum creatinine 0.8 mg/dL (males and females)
• 6 to < 10 years of age: max serum creatinine 1 mg/dL (males and females)
• 10 to < 13 years of age: max serum creatinine 1.2 mg/dL (males and females)
• 13 to < 16 years of age: max serum creatinine 1.5 md/dL (males) and 1.4 md/dL (females)
• >= 16 years of age: max serum creatinine 1.7 mg/dL (males) and 1.4 mg/dL (females)
• The threshold creatinine values were derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the Centers for Disease Control and Prevention (CDC)
• Total or direct bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
• Serum glutamate pyruvate (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (3x ULN); for the purpose of this study, the ULN for SGPT is 45 U/L
• Central nervous system function defined as:
• Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
• Patients must not be in status epilepticus, a coma or on assisted ventilation at the time of study enrollment
• Patients must have receptive and expressive language skills in English, French, or Spanish to complete the QoL and neurocognitive assessments; if a patient meets these criteria but the parent/guardian speaks a language other than English, French, or Spanish, the patient may still be enrolled and tested, and the parent-report measures should be omitted
• All patients and/or their parents or legal guardians must sign a written informed consent; assent, when appropriate, will be obtained according to institutional guidelines
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:

• Patients with metastatic disease by either MRI evaluation (brain and spine) or lumbar CSF cytology are not eligible; patients who are unable to undergo a lumbar puncture for assessment of CSF cytology are ineligible
• Patients must not have received any prior radiation therapy or chemotherapy (tumor-directed therapy) other than surgical intervention and/or corticosteroids
• Pregnancy and Breast Feeding
• Female patients who are pregnant are ineligible due to risks of fetal and teratogenic adverse events as seen in animal/human studies
• Lactating females are not eligible unless they have agreed not to breastfeed their infants
• Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained
• Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation
• Patients with a history of moderate to profound intellectual disability (i.e., intelligence quotient [Q)]=< 55) are not eligible for enrollment; PLEASE NOTE: Children with a prior history of attention deficit hyperactivity disorder (ADHD) or a specific learning disability (e.g., dyslexia) are eligible for this study
Drug: Cisplatin, Drug: Cyclophosphamide, Other: Laboratory Biomarker Analysis, Drug: Lomustine, Radiation: Radiation Therapy, Drug: Vincristine, Drug: Vincristine Sulfate
Medulloblastoma
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The REPLACE Registry

This is a prospective, observational, non-interventional patient registry designed to document product safety and clinical outcomes for 10 years in patients treated with Cholbam/Kolbam, including those who have been using Cholbam/Kolbam for at least one month (existing users) and those who start Cholbam/Kolbam treatment at enrollment (new users). Patients who have been using Cholbam/Kolbam for less than one month and those who had previously been treated and who restart treatment will also be included but will not be counted in the existing or new users groups.

Boris Sudel
All
Not specified
Post Market Monitoring
This study is NOT accepting healthy volunteers
NCT03115086
STUDY00003757
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Inclusion Criteria:

• Male and female patients, of any age.
• The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent, and the patient, if less than 18 years of age, is willing to provide assent as appropriate and in accordance with local regulatory, IRB, and EC requirements.
• The patient has a diagnosis for which Cholbam is indicated.
• The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).
Exclusion Criteria:

• Patients who, by judgement of the Investigator, will not be able to comply with the requirements of the protocol will be excluded
Drug: Cholbam
Bile Acid Synthesis Disorders
Bile Acid Synthesis Disorder, Zellweger Spectrum Disorder, Peroxisomal Disorder, Cholic Acid, Cholbam, The REPLACE Registry
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EnVision CF Multicenter Study of Glucose Tolerance in Cystic Fibrosis

The purpose of this research study is because Cystic Fibrosis Related Diabetes (CFRD) has been identified by the cystic fibrosis (CF) community as one of the top ten priorities for CF research. We know that high blood sugars caused by not enough insulin lead to worse lung function in CF even before diabetes develops. However, we do not know which people with abnormal blood sugars will have long term problems.

Amir Moheet
All
6 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT03650712
STUDY00004854
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Inclusion Criteria:

• Age >/= 6 years
• Diagnosis of cystic fibrosis
• CF patients regularly attending the CF centers
• Clinically stable in previous 3wks:
• absence of major clinical events including pulmonary exacerbations,
• no change in their habitual treatment regimen including introduction of antibiotics or steroids in the past 3 weeks
Exclusion Criteria:

• Diagnosis of type 1 diabetes, type 2 diabetes, or MODY
• Organ transplantation
• new diagnosis of CFRD in the past 6 months
• antidiabetic treatment in past 6 mos (insulin or oral hypoglycemic agents) -patients with previous CFRD diagnosis, but not currently taking insulin/glucose-lowering medications for at least 6 months should be included
• pulmonary exacerbation associated with systemic steroid requirement in the last 6 months
• on CFTR corrector less than 6 months prior to enrollment
Diagnostic Test: Oral glucose tolerance test, Diagnostic Test: Continuous glucose monitoring, Diagnostic Test: Dexa scan
Cystic Fibrosis-related Diabetes
cystic fibrosis, insulin, glucose, children, abnormal glucose tolerance, impaired glucose tolerance, indeterminate glycemia, diabetes
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An Open Label Study to Evaluate DPCP Ointment for the Treatment of Alopecia Areata

This is an open labeled study to determine the response and characteristics, safety and efficacy, of the proprietary DPCP ointment composition as a topical immunotherapeutic agent for the treatment of extensive alopecia areata.

Maria Hordinsky
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03651752
1407M52002
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Inclusion Criteria:

• Subject has clinical diagnosis of extensive alopecia areata (76%-99% involvement as determined by SALT score, Appendix B, Part I).
• Written informed consent and HIPAA authorization have been obtained.
• Subject is > 18 to years of age.
• Female subjects of childbearing potential have a negative pregnancy test and agree to use an acceptable, highly effective method of birth control (i.e., failure rate of less than 1% per year) to prevent pregnancy.
• Subject agrees to comply with protocol requirements and attend all required study visits and is considered to be a good study subject.
• Subject meets concomitant medication washout requirements -
Exclusion Criteria:

• Subject has <76 or greater than 99% hair loss.
• Subject is pregnant or lactating.
• Subject has current controlled or uncontrolled bacterial, viral (with the exception of herpes simplex), fungal, atypical, or opportunistic infection(s).
• Subject has a history of substance abuse within the past five years.
• Immunosuppression (history of transplantation, chemotherapy, splenectomy, HIV).
• Administration of systemic treatment (e.g., Imuran, biologics) that have an immunomodulatory mechanism of action in the preceding 3 months.
• Previous treatment with DPCP.
• Application of topical immunomodulating agent in the preceding 6 weeks.
• Application of topical or intralesional corticosteroids within the past 6 weeks.
• Systemic (oral, inhaled, or intravenous) administration of corticosteroid or other systemic treatment (i.e., prednisone) with an immunosuppressive mechanism of action within the past 3 months.
• Use of light treatments (e.g., PUVA, narrow band UVB) in the preceding 6 weeks.
• Use of Anthralin in preceding 6 weeks.
• Use of minoxidil, topical or oral, in the preceding 4 weeks.
• Subject is currently or has undergone systemic therapy for malignancy within the past five years except for adequately treated Squamous Cell Carcinoma (SCC) or Basal Cell Carcinoma (BCC) of the skin.
• Clinical evidence of secondary skin infection (i.e., folliculitis).
• Participation in other therapeutic investigational clinical trials within 4 weeks of enrollment.
• Evidence of anemia, thyroid disease, sarcoidosis or other medical condition that could be adversely affected by participating in the study.
• Subject has any medical condition that, in the judgment of the Investigator, would jeopardize the subject's safety following exposure to the administered medications. -
Drug: Diphenylcyclopropenone (DPCP) Ointment
Alopecia Areata
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RPC01-3202: INDUCTION STUDY #2 - A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF ORAL OZANIMOD AS INDUCTION THERAPY FOR MODERATELY TO SEVERELY ACTIVE CROHN S DISEASE

This is a Phase 3, randomized, double-blind, placebo-controlled study to determine the effect of oral ozanimod as an induction treatment for subjects with moderately to severely active CD, defined as a CDAI score ≥ 220 to ≤ 450. Approximately 600 subjects with active clinical symptoms and mucosal inflammation will be randomized in a 2:1 ratio to receive either ozanimod or placebo. Subjects will be stratified by prior biologic use and corticosteroid use at baseline. Approximately 50% of subjects with a history of treatment with marketed biologic agents (eg, TNF antagonists, anti-IL-12/23 and anti-integrin therapy) will be recruited. This limit will ensure the enrollment of subjects who have failed or been intolerant to corticosteroids or immunomodulators but never failed a TNF antagonist.

Eugenia Shmidt
All
18 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03440385
STUDY00003227
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For more information regarding Bristol-Myers Squibb Clinical Trial participation, please visit: www.BMSStudyConnect.com
Inclusion Criteria:

• Crohn's disease for ≥ 3 months on endoscopy and on histological exam
• Inadequate response or loss of response to corticosteroids, immunomodulators, and/or biologic therapy
• Crohn's Disease Activity Index (CDAI) score ≥ 220 and ≤ 450
• Average daily stool frequency ≥ 4 points and/or an abdominal pain of ≥ 2 points
• Simple Endoscopic Score for Crohn's Disease (SES-CD) score of ≥ 6 (or SES-CD ≥ 4 in participants with isolated ileal disease)
Exclusion Criteria:

• Diagnosis of ulcerative colitis, indeterminate colitis, radiation colitis, or ischemic colitis, or has strictures with prestenotic dilatation requiring procedural intervention
• Extensive small bowel resection (>100cm) or known diagnosis of short bowel syndrome, or requires total parenteral nutrition
• Current stoma, ileal-anal pouch anastomosis, or fistula Other protocol-defined inclusion/exclusion criteria apply
Drug: Ozanimod, Other: Placebo
Crohn Disease, Digestive & Liver Health
Crohn's Disease, Crohn Disease, Oral, Ozanimod, Moderately active, Severely active, RPC01, RPC01-3202, Clinics and Surgery Center (CSC), inflammatory bowel disease
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Targeting emotional eating and weight loss in adolescents (a.k.a Families, Responsibility, Education, Support, Health for Teens (FRESH-TEEN Study) (FRESH-TEEN)

The two primary aims of the study are: -To evaluate the efficacy of PEER and BWL on the target adolescent’s weight over the 18 months of the study, and -To evaluate the efficacy of PEER and BWL on the target adolescent’s emotion regulation skills and emotional eating over the 18-months of the study.

Carol Peterson
All
13 Years to 16 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03674944
STUDY00003419
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Inclusion Criteria:

• Adolescent age 13-16 years
• BMI percentile 85%85%-99.9% (adolescent only)
• Ability to read English at a minimum 6th grade level (both adolescent and parent); and willing to participate in a 6-month treatment and all assessments (both adolescent and parent)
Exclusion Criteria:

• Current enrollment in a weight management program (Child and Parent);
• Medication that is specifically prescribed for weight loss (Child and Parent);
• Medical or psychiatric condition that may interfere with treatment participation (Child and Parent);
• Regular use of compensatory behavior for weight loss (e.g., purging) during the past six months (Child and Parent);
• Current pregnancy or lactating (Child and Parent);
• Change in psychotropic medication during the previous three months (Child and Parent).
Behavioral: Behavioral Weight Loss (BWL) + Emotion Regulation (ER), Behavioral: Behavioral Weight Loss (BWL)
Overweight and Obesity
Overweight, Obesity, Overeating, Treatment, Body Mass Index, Intervention, Behavioral Treatment, Dialectical Behavior Therapy, Emotion Regulation
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EVALUATION OF PATIENT OUTCOMES FROM THE KIDNEY ALLOGRAFT OUTCOMES ALLOSURE REGISTRY (KOAR) (KOAR)

AlloSure KOAR will recruit eligible kidney recipients who are undergoing AlloSure testing as part of their clinical care to participate in a registry. Patient consent will permit collection and use of data from the patient EMR for entry into the AlloSure database. We will be collecting additional data and outcomes to follow the clinical inquiry and use of AlloSure testing. The AlloSure test has been approved for Medicare coverage for clinical use when a physician determines there is a need to assess the probability of allograft rejection in kidney transplant recipients. The DART study suggests that use of the non-invasive AlloSure test to measure donorderived cell-free DNA (dd-cfDNA) can be used to discriminate active rejection in a renal transplant recipient. Use of the test may reduce invasive percutaneous renal biopsy procedures among patients with a suspicion of rejection. Patients may begin use of the AlloSure test 2 weeks following transplantation. The recommended AlloSure testing schedule is at months 1, 2, 3, 4, 6, 9, and 12 post-transplant and quarterly in year 2 and year 3 post-transplant. AlloSure testing may also be performed when there is clinical suspicion of rejection or other cause of instability in the allograft to inform on the need for a clinically indicated biopsy. Patients and clinicians may choose to use AlloSure testing as a replacement or pre-test for planned surveillance biopsies to inform on the decision to perform a surveillance biopsy, but not the one-year surveillance biopsy used to generate the primary endpoint data. This is a multicenter, non-blinded, prospective observational cohort study of 1000 patients enrolled in an AlloSure testing registry, including 300 patients at centers with planned renal surveillance biopsies at 12 months post-transplantation. The other 700 patients will be from centers that do not perform protocol surveillance biopsies. Outcomes in this sub-cohort, which represents the majority of the intended use population in the U.S., will be compared to the outcomes of the test and control cohorts. A matched control cohort of 300 patients will be retrospectively selected from the subset of centers providing the test cohort patients who have planned surveillance biopsies at 12 months post-transplantation.

Arthur Matas, MD
All
18 Years and over
NA
This study is NOT accepting healthy volunteers
NCT03326076
STUDY00002597
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KOAR
Inclusion Criteria:

• Patient's health care provider adopts and intends to apply the center's AlloSure Routine Testing Schedule as part of the information used to manage the patient.
• Subjects willing to provide written informed consent to participate. KOAR
Exclusion Criteria:
___________________________________________________________ Exclusions for AlloSure® Intended Use Specimens from patients for whom any of the following are true will not be tested:
• Recipients of transplanted organs other than kidney
• Recipients of a transplant from a monozygotic (identical)
• Recipients of a bone marrow transplant
• Recipients who are pregnant
• Recipients who are under the age of 18
• Recipient who are less than 14 days post-transplant
Diagnostic Test: Donor-derived cell-free DNA (AlloSure®), Other: Standard care, Diagnostic Test: Peripheral blood gene expression profiling (AlloMap Kidney), Diagnostic Test: Analytic platform (IBox)
Kidney Transplant Rejection
Clinics and Surgery Center (CSC)
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MT2017-30 :Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant following Reducing Intensity Conditioning for Patients with Selected High Risk Non-Malignant Disease

Christen Ebens
All
up to 25 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03367546
STUDY00001922
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Inclusion Criteria:

• Sickle Cell Disease (SCD) * If diagnosis of SCD must meet one or more of the following disease characteristics:
• Stroke, CNS hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral MRI or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
• Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions
• Recurrent vaso-occlusive pain 3 or more episodes per year for 3 years or more years or recurrent priapism,
• Impaired neuropsychological function and abnormal cerebral MRI scan
• Stage I or II sickle lung disease,
• Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate [GFR] 30-50% of the predicted normal value)
• Bilateral proliferative retinopathy and major visual impairment in at least one eye
• Osteonecrosis of multiple joints with documented destructive changes
• Requirement for chronic transfusions
• RBC alloimmunization
• Transfusion Dependent Alpha- or Beta-Thalassemia
• Other Non-Malignant Hematologic Disorders: Transfusion dependent or involve other potential life-threatening cytopenias, including but not limited to Paroxysmal Nocturnal Hemoglobinuria, Glanzmann's Thrombasthenia, Severe Congenital Neutropenia and Shwachman-Diamond Syndrome
• cALD
• Diagnosis of ALD by abnormal plasma very long chain fatty acid (VLCFA) profile or ABCD1 gene mutation
• Cerebral disease on MRI
• Absence of a Major Functional Disability (cortical blindness, loss of communication, wheelchair dependence) on the ALD Neurologic Function Scale
• Other inherited metabolic disorders: Any other inherited metabolic disorder for which alloHCT is indicated and for whom, in the opinion of the treating physician, the patient's best treatment option is with a haploidentical donor following non-myeloablatve conditioning.
• Age, Performance Status, Consent
• Age: 0-55 years
• Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70
• Consent: voluntary written consent (adult or parental/guardian)
• Adequate Organ Function
• Renal: Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
• Hepatic: Bilirubin and ALT <3 times the upper limit of institutional normal
• Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%.
Exclusion Criteria:

• Availability of a suitable HLA-matched related donor
• Uncontrolled infection
• Pregnant or breastfeeding
• HIV positive
Procedure: Blood and Marrow Transplant
Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
SCD, cALD, Clinics and Surgery Center (CSC)
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MT2018-19: COG ANBL1531 - A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379)

This partially randomized phase III trial studies iobenguane I-131 or ALK Inhibitor Therapy and standard therapy in treating younger patients (365 days to 30 years of age) with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma.

Emily Greengard
All
365 Days to 30 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03126916
STUDY00003568
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Inclusion Criteria:

• Patients must be enrolled on ANBL00B1 (NCT00904241) or APEC14B1 (NCT02402244) prior to enrollment on ANBL1531 (NCT03126916)
• Patient must be >= 365 days and =< 30 years of age at diagnosis
• Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; the following disease groups are eligible:
• Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features:
• MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
• Age > 547 days regardless of biologic features
• Patients with INRG stage MS disease with MYCN amplification
• Patients with INRG stage L2 disease with MYCN amplification
• Patients > 547 days of age initially diagnosed with INRG stage L1, L2 or MS disease who progressed to stage M without prior chemotherapy may enroll within 4 weeks of progression to stage M
• Patients >= 365 days of age initially diagnosed with MYCN amplified INRG stage L1 disease who progress to stage M without systemic therapy may enroll within 4 weeks of progression to stage M
• Patients initially recognized to have high-risk disease must have had no prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing); patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high risk disease but subsequently found to meet the criteria will also be eligible; patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/sex as follows:
• 1 to < 2 years: male = 0.6; female = 0.6
• 2 to < 6 years: male = 0.8; female = 0.8
• 6 to < 10 years: male = 1; female = 1
• 10 to < 13 years: male = 1.2; female = 1.2
• 13 to < 16 years: male = 1.5; female = 1.4
• >= 16 years: male = 1.7; female = 1.4
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN; for the purposes of this study, ULN for SGPT (ALT) is 45
• Shortening fraction of >= 27% by echocardiogram, or ejection fraction of > 50% by echocardiogram or radionuclide angiogram
• No known contraindication to peripheral blood stem cell (PBSC) collection; examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure
Exclusion Criteria:

• Patients with INRG stage L2 tumors without amplification of MYCN regardless of tumor histology (may meet criteria for high risk classification but are not eligible for this trial)
• Patients with bone marrow failure syndromes
• Patients for whom targeted radiopharmaceutical therapy would be contraindicated due to underlying medical disorders
• 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: Autologous Hematopoietic Stem Cell Transplantation, Drug: Busulfan, Drug: Carboplatin, Drug: Cisplatin, Drug: Cyclophosphamide, Drug: Dexrazoxane Hydrochloride, Biological: Dinutuximab, Drug: Doxorubicin Hydrochloride, Drug: Etoposide Phosphate, Radiation: External Beam Radiation Therapy, Radiation: Iobenguane I-131, Drug: Isotretinoin, Drug: Lorlatinib, Drug: Melphalan Hydrochloride, Biological: Sargramostim, Procedure: Therapeutic Conventional Surgery, Drug: Thiotepa, Drug: Topotecan Hydrochloride, Drug: Vincristine Sulfate
Ganglioneuroblastoma, Neuroblastoma
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A Phase II Study of Pembrolizumab Monotherapy in Recurrent Ovarian Cancer of the Immunoreactive Subtype determined by NanoString Gene Expression Profiling

To evaluate clinical anti-tumor activity of pembrolizumab monotherapy based on ORR as assessed by the investigator per irRECIST in patients with ROC whose tumors show an immunoreactive gene expression signature

Boris Winterhoff
Female
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03732950
STUDY00006054
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Inclusion Criteria:

• Be willing and able to provide written informed consent/assent for the trial
• Have received 1-5 prior lines for treating ROC (i.e. 2-6 total prior lines counting the front line) and must have a platinum-free interval (PFI) or a treatment-free interval (TFI) >= 3 months based on the last regimen received
• Have measurable disease based on Response Evaluation Criteria in Solid Tumors (RECIST)
• 1
• Note: Tumor lesions situated in a previously irradiated area are considered measurable if progression has been demonstrated in such lesions
• Have a performance status of 0 or 1 on the Eastern Cooperative Oncology Group (ECOG) performance scale
• Have histologically diagnosed recurrent epithelial ovarian, fallopian or primary peritoneal ovarian cancer
• Have provided a tumor tissue sample either collected from a newly obtained tumor tissue biopsy or an archival tissue specimen. Subjects for whom newly obtained samples cannot be provided (e.g. inaccessible or subject safety concern) may submit an archived biopsy specimen. Formalin-fixed paraffin embedded (FFPE) block specimens are preferred to 20 unstained slides. Additional samples may be requested if tumor tissue provided is not adequate for quality and/or quantity as assessed by the central laboratory
• Performed within 10 days of treatment initiation: absolute neutrophil count (ANC) >= 1,500/mcL
• Performed within 10 days of treatment initiation: platelets >= 100,000/mcL
• Performed within 10 days of treatment initiation: hemoglobin >= 9 g/dL or >= 5.6 mmol/L without transfusion or erythropoietin (EPO) dependency (within 7 days of assessment)
• Performed within 10 days of treatment initiation: serum creatinine OR measured or calculated creatinine clearance (CrCl) (glomerular filtration rate [GFR] can also be used in place of creatinine or CrCl) =< 1.5 x upper limit of normal (ULN) OR >= 45 mL/min for subject with creatinine levels > 1.5 x institutional ULN
• Creatinine clearance should be calculated per institutional standard
• Performed within 10 days of treatment initiation: serum total bilirubin =<1.5 x ULN OR direct bilirubin =< ULN for subjects with total bilirubin levels > 1.5 ULN
• Performed within 10 days of treatment initiation: aspartate aminotransferase (AST) (serum glutamate pyruvate transaminase [SGOT]) and aspartate aminotransferase (ALT) (serum glutamic-oxaloacetic transaminase [SGPT]) =< 2.5 x ULN OR =< 5 x ULN for subjects with liver metastases
• Female subject of childbearing potential should have a negative urine or serum pregnancy within 72 hours prior to receiving the first dose of study medication. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
• Have received front line platinum-based chemotherapy (preoperative chemotherapy is allowed)
• Female subjects of childbearing potential must be willing to use an adequate method of contraception for the course of the study through 120 days after the last dose of study medication
• Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject
Exclusion Criteria:

• Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 2 weeks of the first dose of treatment
• Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of trial treatment
• Has a known history of active TB (Bacillus Tuberculosis)
• Hypersensitivity to pembrolizumab or any of its excipients
• Has had a prior anti-cancer monoclonal antibody (mAb) within 4 weeks prior to study day 1 or who has not recovered (i.e., =< grade 1 or at baseline) from adverse events due to agents administered more than 4 weeks earlier
• Has had prior chemotherapy, targeted small molecule therapy, or radiation therapy within 2 weeks prior to study day 1 or who has not recovered (i.e., =< grade 1 or at baseline) from adverse events due to a previously administered agent
• Note: Subjects with =< grade 2 neuropathy are an exception to this criterion and may qualify for the study
• Note: If subject received major surgery, they must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy
• Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer
• Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis. Subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of trial treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 7 days prior to trial treatment. This exception does not include carcinomatous meningitis which is excluded regardless of clinical stability
• Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (eg., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment
• Has known history of, or any evidence of active, non-infectious pneumonitis
• Has an active infection requiring systemic therapy
• Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject?s participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator
• Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial
• Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment
• Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent
• Has a known history of human immunodeficiency virus (HIV) (HIV 1/2 antibodies)
• Has known active hepatitis B (e.g., hepatitis B surface antigen [HBsAg] reactive) or hepatitis C (e.g., hepatitis C virus [HCV] ribonucleic acid [RNA] [qualitative] is detected)
• Has received a live vaccine within 30 days of planned start of study therapy
• Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist) are live attenuated vaccines, and are not allowed
Biological: Pembrolizumab
Recurrent Fallopian Tube Carcinoma, Recurrent Ovarian Carcinoma, Recurrent Primary Peritoneal Carcinoma
Clinics and Surgery Center (CSC)
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Psychophysiological Stress Reactivity as a Determinant in Co-occurring Alcohol Use and Anxiety Disorders: Diagnosis and Alcohol Use Outcomes

Comorbid AUD+AnxD is a significant barrier to successful AUD treatment. Converging evidence implicates overlap in dysregulation of systems governing stress response (HPA, ANS, CNS) for symptom development in AUD and AnxD. However, this must be systematically demonstrated in comorbid AUD+AnxD. We will assess markers of multi-system biological stress regulation (at rest and in response to laboratory challenge) in alcohol use disorder (AUD) inpatients with and without co-occurring anxiety disorder (AnxD), as well as those with AnxD who do versus do not receive a cognitive behavioral treatment that specifically targets comorbid AUD-AnxD. Laboratory measures include 1) cortisol (to assess the hypothalamic–pituitary–adrenal axis system [HPA] function; 2) heart rate variability (to assess autonomic nervous system [ANS] function), and 3) threat-potentiated startle (to assess central nervous system [CNS] function). Laboratory assessments will occur at the following times: 1) shortly after AUD treatment admittance (Visit 2: Pre-Treatment), 2) immediately following the 4-week AUD treatment (Visit 3: Post-Treatment), 3) 1 month following AUD treatment (Visit 4: 1-Month Follow-Up), and 4) 4 months following AUD treatment (Visit 5: 4-Month Follow-Up). Self-reported alcohol intake will be assessed at baseline as well as at the 1- and 4-Month Follow-Ups to determine whether laboratory stress measures predict treatment outcomes. A single laboratory assessment of healthy controls will serve as a normative reference for characterizing patient laboratory responses in terms of dysregulation and re-regulation.

Justin Anker
All
18 Years to 65 Years old
NA
This study is also accepting healthy volunteers
NCT03056872
1612M02641
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Inclusion Criteria:

• Ability to provide informed consent
• Between the ages of 18 and 65
• Diagnostic and Statistical Manual diagnosis of a Panic Disorder, Generalized Anxiety Disorder, or Social Anxiety Disorder within the past 30 days (AUD+AnxD group only).
• Primary alcohol use disorder diagnosis and alcohol use in the 30 days preceding the study (AUD alone and AUD+AnxD groups only).
• Inpatient treatment at Lodging Plus primarily for alcohol (vs. other drug with nicotine accepted) dependence (AUD alone and AUD+AnxD groups only).
• A minimum of a sixth-grade reading level.
• Healthy controls, same criteria absent AUD and AnxD diagnosis of an alcohol and/or anxiety disorder
• Lives within proximity to the Twin Cities (e.g., within about an hour's drive) or willing to drive to Fairview for the purpose of attending follow-up visits
• Willingness to provide contact information to confirm follow-up appointments
Exclusion Criteria:

• Lifetime history of psychosis or mania
• Cognitive impairment, physical impairment, or chronic medical illness that precludes study participation
• Primary PTSD as determined by qualifying assessment
• Females currently pregnant
• Exposure to antipsychotic medication for a total duration >16 weeks.
• Prior head injury leading to >30 minutes of unconsciousness.
• Cognitive impairment that impedes study participation.
• Healthy controls with a history of any major medical or psychiatric disorders (e.g., schizophrenia, depression, heart disease, or stroke).
• Suicide intent or attempt in the past 30 days
• Cardiovascular health issues
• Thyroid Disease
• History of severe neurological illness such as chronic seizure disorder (e.g, epilepsy) or stroke
• Brain tumor and/or implants in the skull cavity (e.g., plate in the skull)
• Pacemaker
Alcohol Use Disorder, Anxiety Disorder/Anxiety State, Stress Disorder, Hypothalamic Pituitary Adrenal, Drinking to Cope
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Vertical Sleeve Gastrectomy and Lifestyle Modification for the Treatment of Non-Alcoholic Steatohepatitis

Participants patients meeting study entry criteria are randomized with equal probability to one of two study groups: (1) Lifestyle modification or (2) Vertical Sleeve Gastrectomy (VSG) with Iifestyle modification, followed for 12 months. The primary goal for the trial is to determine if we can recruit, randomize, and retain participants to perform invasive and non-invasive measurements of NASH and fibrosis, deliver lifestyle modification and demonstrate safety of VSG. We wish to also understand which of these two interventions is more effective in achieving, 12 months after entry into the trial, a reduction in NAS composed of the non-weighted scores: (1) steatosis 0-3 (2) Inflammation 0-3 and (3) ballooning 0-2. Secondary goals include comparing the two treatment groups for changes in other measured outcomes including MRI assessments of intrahepatic triglyceride and liver elasticity and serum markers. As a pilot study a sample size of 19 in each group should offer significant information as to the difference in NAS score reduction between to two groups and to achieve adequate power to distinguish clinically significant changes in the primary and secondary outcome measures. These data support the overarching objective i.e. to provide evidence that a larger, longer-term clinical outcomes trial is feasible. A goal is for a longer term follow up for 5 years to assess the durability of treatment effects and treatment differences.

Sayeed Ikramuddin
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT03587831
STUDY0002886
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Inclusion Criteria:

• Age 40 to 67 years at eligibility visit.
• At least one of the following: A) Liver biopsy within 6 months preceding enrollment consistent with non-alcoholic fatty liver disease with a NAFLD Activity Score ≥4 B) Diagnosed with type 2 diabetes mellitus or prediabetes for at least 6 months prior to enrollment, under the active care of a doctor for at least the six months prior to enrollment, HbA1c< 9% and NOT requiring insulin
• Body Mass Index (BMI): 35.0-50.0 kg/m2 at eligibility visit
• Willingness to accept random assignment to either treatment group
• All patients must have insurance with no exclusion for obesity related treatments or management of obesity surgery complications. This applies to all participants enrolled in the study
• Expect to live or work within approximately two-hours traveling time from the study clinic for the duration of the one-year trial
• Evidence of liver fat present in the baseline MR images
• Suitable for liver biopsy using the percutaneous approach
• Willingness to comply with the follow-up protocol and successful completion of the run-in (described below)
• Written informed consent
Exclusion Criteria:

• Cardiovascular event (myocardial infarction, acute coronary syndrome, coronary artery angioplasty or bypass, stroke) in the past six months.
• Current evidence of congestive heart failure, angina pectoris, or symptomatic peripheral vascular disease.
• Pulmonary embolus or thrombophlebitis in the past six months.
• Cancer of any kind (except basal cell skin cancer or cancer in situ) unless documented to be disease-free for five years.
• Significant anemia (hemoglobin 1.0 g/dL or more below normal range) or history of coagulopathy.
• Serum creatinine >1.5 mg/dL.
• Serum total bilirubin greater than the upper limit of normal in the absence of Gilbert's syndrome, or alkaline phosphatase or ALT or AST greater than twice the upper limit of normal. Elevated INR.
• Alcohol intake more than one drink or >20 grams per day
• History of stomach surgery, bile duct surgery, pancreatic surgery, splenectomy, or colon resection.
• Gastric or duodenal ulcer in the past six months.
• History of intra-abdominal sepsis (except for uncomplicated appendicitis or diverticulitis more than six months prior to enrollment).
• Previous organ transplantation.
• Self-reported HIV-positive status, active tuberculosis, active malaria, chronic hepatitis B or C, cirrhosis, or inflammatory bowel disease.
• Currently pregnant or nursing, or planning to become pregnant in the next two years.
• History of alcohol, drug, or opioid dependency (excluding nicotine) in the past five years.
• Active psychosocial or psychiatric problem that is likely to interfere with adherence to the protocol.
• Brief psychological evaluation recommendation that individual not continue in the study.
• Current participation in a conflicting research protocol.
• Presence of any chronic or debilitating disease that would make adherence to the protocol difficult.
• Serum c-peptide <1.0 ng/ml post prandial.
• Exclusions may also be made at the discretion of the attending physician or the eligibility committee.
• Contraindication to MRI scanning. MRI contraindications are assessed during initial eligibility review as well as on the day of scanning using the standard safety screening form.
• Individuals that require the trans-jugular approach for liver biopsy
• Gastroesophageal reflux disease requiring medications. History of endoscopy demonstrating esophagitis or Barrett's changes in the esophagus. Any history of dysphagia.
• More than 2 cups of coffee per day
• NAS fibrosis score > 3
Procedure: Vertical Sleeve Gastrectomy, Behavioral: Lifestyle Modification Counseling
Digestive & Liver Health, NASH - Nonalcoholic Steatohepatitis
Clinics and Surgery Center (CSC), Bariatric Surgery, NASH, VSG
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RPC01-3203: A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study of Oral Ozanimod as Maintenance Therapy for Moderately to Severely Active Chron's Disease

This is a Phase 3, randomized, double-blind, placebo-controlled study to demonstrate the effect of oral ozanimod as maintenance therapy in subjects with moderately to severely active CD, defined as a CDAI score of ≥ 220 to ≤ 450. Subjects who complete the initial 12 weeks of treatment (Induction Studies RPC01-3201 or RPC01-3202) and are in clinical response (CDAI reduction from baseline of ≥ 100 points or CDAI score of < 150) and/or clinical remission (CDAI score < 150 and/or average stool frequency score ≤ 3 with a stool frequency no worse than baseline and an average abdominal pain score ≤ 1) will be eligible to participate in the Maintenance Study.

Eugenia Shmidt
All
18 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03464097
STUDY00003228
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Inclusion Criteria:

• Fulfilled the inclusion criteria at time of entry into the induction study and completed the week 12 efficacy assessments of the induction study
• In clinical response and/or clinical remission and/or an average daily stool frequency score ≤ 3 and an average abdominal pain score ≤ 1 with abdominal pain and stool frequency no worse than baseline at Week 12 of the Induction Study
Exclusion Criteria:

• Partial or total colectomy, small bowel resection, or an ostomy since day 1 of the induction studies or has developed a symptomatic fistula
• Had a rectal steroid therapy, rectal 5-aminosalicylates, parenteral corticosteroids, immunomodulatory agents, investigational agents or apheresis Other protocol-defined inclusion/exclusion criteria apply
Drug: Ozanimod, Other: Placebo
Crohn Disease, Digestive & Liver Health
Crohn's Disease, Crohn Disease, Oral, Ozanimod, Moderately active, Severely active, RPC01, RPC01-3203, Clinics and Surgery Center (CSC), inflammatory bowel disease
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Laryngeal Vibration for Spasmodic Dysphonia (SD-VTS)

All
18 Years to 75 Years old
N/A
This study is also accepting healthy volunteers
NCT03746509
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Inclusion Criteria:

• diagnosis of adductor SD for a minimum of 6 months with documented symptom relief after botox injection
Exclusion Criteria:

• abductor SD
• patients with other voice disorders such as muscle tension dysphonia that share some of the symptomology with SD
Device: Laryngeal Vibration (Treatment), Device: Laryngeal Vibration (Comparator)
Spasmodic Dysphonia
Dystonia, Spasmodic dysphonia, Laryngeal dystonia
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MT2017-17: T cell receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients with Fanconi Anemia (FA)

The primary objective is to determine the incidence of grade II-IV acute graft versus host disease (GVHD) by Day 100 using an alpha/beta T cell depleted peripheral blood stem cells (PBSC) and without routine GVHD prophylaxis.

Margaret MacMillan, MD
All
up to 65 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03579875
STUDY00003182
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Patient Selection:
Inclusion Criteria:

• Diagnosis of Fanconi anemia
• Less than 65 years of age
• Karnofsky performance status of ≥ 70% or, for children < 16 years of age, Lansky Play Score ≥ 50
• Presence of at least one of the following risk factors:
• Severe aplastic anemia (SAA) defined as: Aplastic anemia is defined as having at least one of the following when not receiving growth factors or transfusions:
• platelet count <20 x 109/L
• absolute neutrophil count of <5 x 108/L
• hemoglobin <8 g/dL
• Myelodysplastic syndrome (MDS) or acute leukemia
• High risk genotype
• Adequate organ function defined as:
• Bilirubin, AST or ALT, ALP <5 x normal, Cardiac: left ventricle ejection fraction (LEFV) ≥45% by ECHO
• Pulmonary: DLCO, FEV1, FVC ≥ 40% predicted, and absence of O2 requirements. For children that are not able to cooperate with PFTs, a pulse oximetry with exercise should be attempted. If neither test can be obtained it should be clearly stated in the physician's note.
• Identification of a suitable donor for peripheral blood cells per match criteria found in Section 5.
• Females of childbearing potential and males with partners of child-bearing potential must agree to use of contraception for the duration of treatment and 4 months after the transplant
• Able to provide written voluntary consent prior to the performance of any research related tests or procedures with parental/guardian consent for minor (and assent as appropriate)
Exclusion Criteria:

• Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration
• Active, uncontrolled infection within 1 week prior to starting study therapy
• Malignant solid tumor cancer within previous 2 years Donor Selection (Inclusion Criteria): meets one of the following match criteria:
• an HLA-A, B, DRB1 matched sibling donor (matched sibling)
• an HLA-A, B, DRB1 matched related donor (other than sibling)
• a related donor mismatched at 1 HLA-A, B, C and DRB1 antigen
• 7-8/8 HLA-A,B,C,DRB1 allele matched unrelated donor per current institutional guidelines Patients and donors are typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing. If a donor has been selected on the basis of HLA-A, B, C and DRB1 typing as above, preference will be made for donors matched at the HLA-C locus.
• Body weight of at least 40 kilograms and at least 12 years of age
• Willing and able to undergo mobilized peripheral blood apheresis
• In general good health as determined by the medical provider
• Adequate organ function defined as:
• Hematologic: hemoglobin, WBC, platelet within 10% of upper and lower limit of normal range of test (gender based for hemoglobin)
• Hepatic: ALT < 2 x upper limit of normal
• Renal: serum creatinine < 1.8 mg/dl
• Performance of a donor infectious disease screen panel including CMV Antibody, Hepatitis B Surface Antigen, Hepatitis B Core Antibody, Hepatitis C Antibody, HIV 1/2 Antibody, HTLVA 1/2 Antibody, Treponema, and Trypanosoma Cruzi (T. Cruzi) plus HBV, HCV, WNV, HIV by nucleic acid testing (NAT); and screening for evidence of and risks factors for infection with Zika virus, or per current standard institutional donor screen - must be negative for HIV and active hepatitis B
• Not pregnant - females of childbearing potential must have a negative pregnancy test within 7 days of mobilization start
• Voluntary written consent (parent/guardian and minor assent, if < 18 years) prior to the performance of any research related procedure
Drug: Total Body Irradiation (TBI) (Plan 1), Drug: Cyclophosphamide (CY) (Plan 1), Drug: Fludarabine (FLU), Drug: Methylprednisolone (MP), Device: Donor mobilized PBSC infusion, Drug: G-CSF, Drug: Cyclophosphamide (CY) (Plan 2), Drug: Rituximab, Drug: Busulfan
Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes
Clinics and Surgery Center (CSC)
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RPC01-3204: A PHASE 3, MULTICENTER, OPEN-LABEL EXTENSION STUDY OF ORAL OZANIMOD FOR MODERATELY TO SEVERELY ACTIVE CROHN S DISEASE

This is a Phase 3, open-label, multicenter extension study to evaluate safety and efficacy of ozanimod in subjects with moderately to severely active CD. Approximately 1200 subjects who have previously participated in a study of ozanimod for CD will be eligible to participate in this study if they meet the eligibility criteria as outlined in the prior study (eg, RPC01-3201, RPC01-3202, RPC01-3203, or RPC01-2201).

Eugenia Shmidt
All
18 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03467958
STUDY00003229
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Inclusion Criteria:

• Is not in clinical response or clinical remission after completing 12 weeks in the Induction Studies
• Experience relapse or who complete the Maintenance Study
• Complete a study of ozanimod for Crohn's Disease and meet the criteria for participation
Exclusion Criteria:

• Has any clinically relevant hepatic, neurological, pulmonary, ophthalmological, endocrine, psychiatric, or other major systemic disease making implementation of the protocol or interpretation of the study difficult or that would put the subject at risk by participating in the study
• Has suspected or diagnosed intra-abdominal or perianal abscess that has not been appropriately treated
• Is receiving treatment with any of the following drugs or interventions within the CYP2C8 inhibitors, inducers and Monoamine oxidase inhibitors Other protocol-defined inclusion/exclusion criteria apply
Drug: Ozanimod
Digestive & Liver Health, Crohn Disease
inflammatory bowel disease, Crohn's Disease, Crohn Disease, Oral, Ozanimod, Moderately active, Severely active, RPC01, RPC01-3204, Clinics and Surgery Center (CSC)
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Neuromodulation and Cognitive Training in Opioid Use Disorder

Patients will receive 20 minutes of continuous stimulation of either active or sham transcranial direct current stimulation every day for five consecutive days. This will allow investigation to whether the pairing of dorsolateral prefrontal corex stimulation and cognitive flexibility training can enhance functional connectivity between dorsolateral prefrontal corex and nucleaus accumbens, assisting with treatment for opioid use disorder.

Jazmin Camchong
All
18 Years to 60 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03773523
STUDY00005047
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Inclusion Criteria:

• 20 abstinent individuals (18-60 years old; 1-2 weeks of abstinence) who meet DSM-V criteria for opioid use disorder (OUD) will be recruited from the Lodging Plus Program, part of University of Minnesota Medical Center
• This 28-day program provides a supervised environment to treat individuals with OUD in which patients receive random drug screenings. Lodging Plus has 50 beds and admits an average of 20 patients per week and about 50% of patients admitted have a diagnosis of opioid use disorder.
• Ability to provide written consent and comply with study procedures, meet the Diagnostic and Statistical Manual of Mental Disorders (DSM-V) diagnostic criteria for OUD. Subjects may have current comorbid drug use, but their primary substance use disorder diagnosis needs to be based on opioid use. Subjects must have the intention to remain in the Lodging Plus program (4 weeks) until the end of the intervention portion of the study. Vulnerable populations will not be included.
Exclusion Criteria:

• Any medical condition or treatment with neurological sequelae (i.e. stroke, tumor, loss of consciousness>30 min, HIV)
• A head injury resulting in a skull fracture or a loss of consciousness exceeding 30 minutes (i.e., moderate or severe TBI)
• Any contraindications for tDCS or MRI scanning (tDCS contraindication: history of seizures; MRI contraindications; metal implants, pacemakers or any other implanted electrical device, injury with metal, braces, dental implants, non-removable body piercings, pregnancy, breathing or moving disorder)
• DSM-V criteria for psychiatric disorder, may have a lifetime diagnosis of depression
• Presence of a condition that would render study measures difficult or impossible to administer or interpret
• Age outside the range of 18 to 60
• Primary current substance use disorder diagnosis on a substance other than opioid except for caffeine or nicotine
• Clinical evidence for Wernicke-Korsakoff syndrome
• Nicotine use will be recorded.
Device: Transcranial Direct Current Stimulation (tDCS)
Opioid-use Disorder, Opioid Dependence, Opioid Abuse
tDCS, Transcranial Direct Current Stimulation, MRI, Opioid dependence, Opioids, Buprenorphine, Suboxone, Lodging Plus, Fairview Lodging Plus
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PANOVA-3: Pivotal, randomized, open-label study of Tumor Treating Fields (TTFields, 150kHz) concomitant with gemcitabine and nab-paclitaxel for front-line treatment of locally-advanced pancreatic adenocarcinoma

Adult patients with locally advanced, unresectable pancreatic carcinomas will be screened at baseline, and randomized 1:1 to treatment in either of two arms: standard-of-care palliative chemotherapy using gemcitabine + nab-paclitaxel (given on days 1, 8, and 15 of each 28-day cycle), or the same chemotherapeutic regimen with the addition of alternating electric tumor-treating fields (TTFields) applied to the patients abdominal cavity using pads attached to a source pack. The TTFields are applied 24 hours per day/7 days per week. Follow-up CT scans will be performed every 8 weeks, as is standard of care, with post-progression follow-up visits and survival follow-up visits every four weeks.

Emil Lou
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03377491
STUDY00004177
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Inclusion Criteria:

• 18 years of age and older
• Life expectancy of ≥ 3 months
• Histological/cytological diagnosis of de novo adenocarcinoma of the pancreas
• Unresectable, locally advanced stage disease according to the following criteria:
• Head/uncinate process:
• Solid tumor contact with SMA>180°
• Solid tumor contact with the CA>180°
• Solid tumor contact with the first jejunal SMA branch
• Unreconstructible SMV/PV due to tumor involvement or occlusion (can be d/t tumor or bland thrombus)
• Contact with most proximal draining jejunal branch into SMV
• Body and tail
• Solid tumor contact of >180° with the SMA or CA
• Solid tumor contact with the CA and aortic involvement
• Unreconstructible SMV/PV due to tumor involvement or occlusion (can be d/t tumor or bland thrombus)
• No distant metastasis, including non-regional lymph node metastasis
• No borderline resectable (per Al-Hawary MM, et al., Radiology 201414)
• ECOG score 0-2
• Amenable and assigned by the investigator to receive therapy with gemcitabine and nab-paclitaxel
• Able to operate the NovoTTF-100L(P) System independently or with the help of a caregiver
• Signed informed consent form for the study protocol
Exclusion Criteria:

• Prior palliative treatment (e.g. surgery, radiation) to the tumor
• Cancer requiring anti-tumor treatment within the 5 years before inclusion, excluding treated stage I prostate cancer, in situ cervical or uterus cancer, in situ breast cancer and non-melanomatous skin cancer.
• Serious co-morbidities:
• Clinically significant (as determined by the investigator) hematological, hepatic and renal dysfunction, defined as: Neutrophil count < 1.5 x 10^9/L and platelet count < 100 x 10^9/L; bilirubin > 1.5 x Upper Limit of Normal (ULN); AST and/or ALT > 2.5 x ULN; and serum creatinine > 1.5 x ULN.
• History of significant cardiovascular disease unless the disease is well controlled. Significant cardiac disease includes second/third degree heart block; significant ischemic heart disease; poorly controlled hypertension; congestive heart failure of the New York Heart Association (NYHA) Class II or worse (slight limitation of physical activity; comfortable at rest, but ordinary activity results in fatigue, palpitation or dyspnea).
• History of arrhythmia that is symptomatic or requires treatment. Patients with atrial fibrillation or flutter controlled by medication are not excluded from participation in the trial.
• History of cerebrovascular accident (CVA) within 6 months prior to randomization or that is not stable.
• Active infection or serious underlying medical condition that would impair the ability of the patient to receive protocol therapy.
• History of any psychiatric condition that might impair patient's ability to understand or comply with the requirements of the study or to provide consent.
• Concurrent anti-tumor therapy beyond gemcitabine and nab-paclitaxel
• Implantable electronic medical devices in the torso, such as pacemakers
• Known severe hypersensitivities to medical adhesives or hydrogel, or to one of the chemotherapies used in this trial.
• Pregnancy or breast-feeding (female patients with reproductive potential and their partners must accept to use effective contraception throughout the entire study period and for 3 months after the end of treatment). All patients who are capable of becoming pregnant must take a pregnancy test which is negative within 72 hours before beginning treatment. The definition of effective contraception is left up to the decision of the investigator.
• Unable to follow the protocol for medical, psychological, familial, geographic or other reasons.
• Admitted to an institution by administrative or court order.
Device: NovoTTF-100L(P), Drug: Gemcitabine, Drug: nab paclitaxel
Pancreas Adenocarcinoma
Locally Advanced Pancreatic Adenocarcinoma, TTFields, Tumor Treating Fields, Gemcitabine, nab-Paclitaxel, Minimal toxicity, TTF, Novocure, Clinics and Surgery Center (CSC)
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COG AGCT1531 - A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors

This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Emily Greengard
All
Not specified
Phase 3
This study is NOT accepting healthy volunteers
NCT03067181
STUDY00003824
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Inclusion Criteria:

• There is no age limit for the low risk stratum (stage I ovarian immature teratoma and stage I non-seminoma or seminoma malignant GCT [all sites])
• Standard risk 1: Patient must be < 11 years of age at enrollment
• Standard risk 2: Patients must be >= 11 and < 25 years of age at enrollment
• Patients enrolling on one of the low risk arms must be newly diagnosed with a stage I germ cell tumor; for the standard risk arms, patients must be newly diagnosed with metastatic germ cell tumor (stage II or higher); histologic confirmation of a primary extracranial germ cell tumor in any of the categories outlined below is required of all patients at enrollment except for those who were initially diagnosed with stage I non-seminoma malignant GCT and later recur during observation post surgery off study; for these patients, if elevated tumor markers rise to > 5 x upper limit of normal (ULN) on at least 2 measurements taken at least 1 week apart, a diagnostic biopsy is not required for enrollment
• Low risk stage I immature teratoma (IT); site: ovarian; stage: Children's Oncology Group (COG) stage I, Federation of Gynecology and Obstetrics (FIGO) stage IA and IB; grade: 2 or 3; histology: pure immature teratoma (may contain microscopic foci of yolk sac tumor), mixed immature and mature teratoma, (no pathological evidence of MGCT); tumor markers: alpha-FP =< 1,000 ng/mL, beta-HCG institutional normal; all ages
• Low risk stage I non-seminoma MGCT; site: ovarian, testicular, or extragonadal; stage: COG stage I, FIGO stage IA and IB, American Joint Committee on Cancer (AJCC) testicular stage IA, IB and IS; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma (pure or mixed); all ages
• Low risk stage I seminoma-MGCT; site: testicular; stage: COG stage I; AJCC testicular stage IA IB, and IS; histology: must contain at least one of the following: may contain immature/mature teratoma; may NOT contain yolk sac tumor, embryonal carcinoma, or choriocarcinoma; all ages
• Standard risk 1 (SR1); site: ovarian, testicular, or extragonadal; stage: COG stage II-IV, FIGO stage IC, FIGO stage II-IV (International Germ Cell Consensus Classification [IGCCC] criteria DO NOT apply); histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) < 11
• Standard risk 2 (SR2)
• Site: ovarian; stage: COG stage II and III, FIGO stage IC, II and III; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) >= 11 and < 25
• Site: testicular; stage: COG stage II-IV, AJCC stage II, III, IGCCC good risk; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; tumor markers: must be IGCCC good risk; post op: alpha-FP < 1,000 ng/mL, beta-HCG < 5,000 IU/mL and lactate dehydrogenase (LDH) <
• 0 x normal; age (years) >= 11 and < 25
• Site: extragonadal; stage: COG stage II; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) >= 11 and < 25
• Notes:
• IGCCC criteria only apply to SR2 patients with a testicular primary tumor
• Use post-op tumor marker levels to determine IGCCC risk group
• Stage 1 seminoma patients are not eligible for the standard risk arms of the study
• For the low risk stage I non-seminoma MGCT and the standard risk arms, components of yolk sac tumor, embryonal carcinoma, or choriocarcinoma can be mixed with other forms of GCT, such as seminoma or mature or immature teratoma; if yolk sac tumor is the only malignant component present, then it must be deemed by the pathologist to be greater than a "microscopic component" of yolk sac tumor
• Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, 2 or 3; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
• Organ function requirements apply ONLY to patients who will receive chemotherapy (SR1 and SR2 patients)
• Adequate renal function defined as:
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 OR
• A serum creatinine based on age/gender as follows: (mg/dL)
• 1 month to < 6 months male: 0.4 female: 0.4
• 6 months to < 1 year male: 0.5 female: 0.5
• 1 to < 2 years male: 0.6 female: 0.6
• 2 to < 6 years male: 0.8 female: 0.8
• 6 to < 10 years male: 1 female: 1
• 10 to < 13 years male: 1.2 female: 1.2
• 13 to < 16 years: male: 1.5 female: 1.4
• >= 16 years male: 1.7 female: 1.4
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
• 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 (for the purpose of this study, the ULN for SGPT is 45 U/L)
• Peripheral absolute neutrophil count (ANC) >= 1,000/mm^3 AND
• Platelet count >= 100,000/mm^3
• Patients enrolling on the standard risk arms must be medically fit to receive protocol treatment and with no contraindications to protocol treatment
• Eligibility criteria to participate in the pilot study of the AYA-Hears instrument (patient reported outcomes [PROs] of ototoxicity) Note: participants in group 1 will not receive AGCT1531 protocol-directed therapy; all other AYA-HEARS patients must be enrolled on the AGCT1531 SR2 arm in order to participate
• >= 11 and < 25 years old at enrollment
• Able to fluently speak and read English
• Has received prior cisplatin- or carboplatin-based chemotherapy regimen for malignancy including diagnoses other than germ cell tumor
• Followed for cancer or survivorship care at one of the following institutions:
• Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
• Dana Farber/Harvard Cancer Center
• Hospital for Sick Children
• Children's Hospital of Eastern Ontario
• Oregon Health and Science University
• Seattle Children's Hospital
• Yale University
Exclusion Criteria:

• Patients with any diagnoses not listed including:
• Stage I testicular cancer patients who have undergone primary RPLND (retroperitoneal lymph node dissection)
• Pure dysgerminoma
• Pure mature teratoma
• Pure immature teratoma COG stage I, grade I
• Pure immature teratoma COG stage I, grade 2,3 with alpha-fetoprotein (AFP) >= 1000 ng/mL
• Pure immature teratoma COG stage II - IV or FIGO stage IC to IV
• "Poor risk" GCT (age >= 11 years old and COG stage IV ovarian, COG stage III or IV EG, or IGCCC intermediate or poor risk testicular), or
• Primary central nervous system (CNS) germ cell tumor
• Germ cell tumor with somatic malignant transformation
• Spermatocytic seminoma
• Patients must have had no prior systemic therapy for the current cancer diagnosis
• Patients must have had no prior radiation therapy with the exception of CNS irradiation of brain metastases; (this exception only applies to SR1 patients; any patients over age 11 with distant metastases to brain [stage IV disease] would be considered poor risk and therefore not eligible for this trial)
• Patients with significant, pre-existing co-morbid respiratory disease that contraindicate the use of bleomycin are ineligible for the standard risk arms of the trial
• 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; (this criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2 patients])
• Lactating females who plan to breastfeed their infants; (this criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2 patients])
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation; (this criteria applies ONLY to patients who will receive chemotherapy [SR1 and SR2 patients])
Other: Best Practice, Biological: Bleomycin Sulfate, Drug: Carboplatin, Drug: Cisplatin, Drug: Etoposide, Other: Laboratory Biomarker Analysis, Other: Pharmacogenomic Study, Other: Quality-of-Life Assessment, Other: Questionnaire Administration
Childhood Extracranial Germ Cell Tumor, Extragonadal Embryonal Carcinoma, Germ Cell Tumor, Malignant Germ Cell Tumor, Malignant Ovarian Teratoma, Stage I Ovarian Choriocarcinoma, Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage I Ovarian Teratoma AJCC v6 and v7, Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage I Testicular Choriocarcinoma AJCC v6 and v7, Stage I Testicular Embryonal Carcinoma AJCC v6 and v7, Stage I Testicular Seminoma AJCC v6 and v7, Stage I Testicular Yolk Sac Tumor AJCC v6 and v7, Stage II Ovarian Choriocarcinoma, Stage II Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage II Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage II Testicular Choriocarcinoma AJCC v6 and v7, Stage II Testicular Embryonal Carcinoma AJCC v6 and v7, Stage II Testicular Yolk Sac Tumor AJCC v6 and v7, Stage III Ovarian Choriocarcinoma, Stage III Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage III Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage III Testicular Choriocarcinoma AJCC v6 and v7, Stage III Testicular Embryonal Carcinoma AJCC v6 and v7, Stage III Testicular Yolk Sac Tumor AJCC v6 and v7, Stage IV Ovarian Choriocarcinoma, Stage IV Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage IV Ovarian Yolk Sac Tumor AJCC v6 and v7, Testicular Mixed Choriocarcinoma and Embryonal Carcinoma, Testicular Mixed Choriocarcinoma and Teratoma, Testicular Mixed Choriocarcinoma and Yolk Sac Tumor
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MT2017-45 :Chimeric Antigen Receptor (CAR)-T Cell Therapy for Patients with Hematologic Malignancies

This is a phase II study of FDA-approved CAR-T products for patients with hematologic malignancies. The study provides criteria for consistent treatment and management according to FDA labelling of CAR-T products and does not contain experimental components. Patients will be assigned to Arms A B and C based on age, CAR-T product and diagnosis. Overall remission rate, safety events and other endpoints will be calculated for Arms A B and C separately.

Veronika Bachanova, MD
All
Not specified
Phase 2
This study is NOT accepting healthy volunteers
NCT03642626
STUDY00004096
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ARM A: Kymriah for Refractory/relapsed B-cell acute lymphoblastic leukemia expressing CD19
Inclusion Criteria:

• Age and Disease Status
• Must be age 0-25 years
• Disease status: Relapsed and refractory pediatric B-cell ALL defined by one of these:
• Primary induction failure with no complete remission after ≥2 cycles of induction chemotherapy, or
• Patients with persistent minimal residual disease (MRD >0.01% by flow cytometry or persistent by cytogenetic or molecular assays) after ≥2 cycles of consolidation chemotherapy, or
• Patients in 2nd or greater relapse of B-ALL or
• Patients with persistent CNS leukemia, or
• Down Syndrome or other congenital diseases assuming that they fit the criteria for second or greater relapse or refractory leukemia, or
• Patients with Ph+ ALL are eligible if theywho have failed or are intolerant to two lines of TKI assuming they fit the criteria for second or greater relapse or are considered refractory.
• Performance Status
• Karnofsky (age ≥16 years) or Lansky (age < 16 years) performance status ≥ 50% at screening
• ALC >500/uL at screening (prior to apheresis) and absolute lymphocyte count >/= 150/uL
• Organ Function
• Renal function defined as:
• A serum creatinine of ≤1.5 x ULN OR
• eGFR ≥ 50 mL/min/1.73 m2
• Liver function defined as: ** ALT ≤ 5 times the ULN for age (unless due to disease) ** Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤
• 5 x ULN
• Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
• Hemodynamically stable and LVEF ≥ 45% confirmed by echocardiogram or MUGA
• Other Inclusion Criteria
• Life expectancy ≥12 weeks
• Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment.
• Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:

• Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
• Patients with Burkitt's lymphoma/leukemia (i.e. patients with mature B-cell ALL, leukemia with B-cell [sIg positive and kappa or lambda restricted positivity] ALL, with FAB L3 morphology and /or a MYC translocation)
• CNS 2A
• CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
• Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
• Uncontrolled active hepatitis B or hepatitis C
• Active HIV infection
• Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
• Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
• Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
• Intolerance to the excipients of the CAR-T cell product
• Any immunosuppressive medication must be stopped ≥ 2 weeks prior to enrollment.
• Patient has taken one of the prohibited concomitant medications within the timeframe outlined in section 6.1 ARM B: Yescarta for Relapsed or Refractory diffuse large B cell lymphoma
Inclusion Criteria:

• Age and Disease Status
• Adult patients (age ≥ 18 years)Patients must be ≥18 years of age
• One of the following histologies and expression of CD19 by tumor cells: ** diffuse large B-cell lymphoma (DLBCL) not otherwise specified, or ** primary mediastinal large B-cell lymphoma, or ** high grade B-cell lymphoma, or ** DLBCL arising from follicular lymphoma
• Disease status: ** Chemotherapy refractory disease after ≥2 lines of chemotherapy, or ** Relapsed with no remission after ≥1 lines of salvage chemotherapy, or ** Relapsed following autologous HCT (and failed at least 2 prior lines of therapy including high dose chemotherapy). If salvage therapy is given post autoHCT, the subject must have no response or relapse after the last line of therapy
• Measurable disease at time of apheresis: Nodal lesions or extranodal lesion
• ECOG performance status 0-2
• ALC >/=100/uL at screening (prior to apheresis)
• Renal function defined as: ** A serum creatinine of ≤1.5 x ULN OR ** eGFR ≥ 50 mL/min/1.73 m2
• Liver function defined as:
• ALT ≤ 5 times the ULN for age (unless due to disease)
• Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN
• Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
• Hemodynamically stable and LVEF ≥ 45% confirmed by echocardiogram or MUGA
• Adequate bone marrow reserve (unless marrow infiltrated by disease) defined as :
• Absolute neutrophil count (ANC) > 1.000/mm3 (only for NHL)
• Platelets ≥ 50.000/mm3 (transfusion support can be provided)
• Hemoglobin >8.0 mg/dl (transfusion support can be provided)
• Life expectancy ≥12 weeks
• Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment.
• Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:

• Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
• Active CNS involvement by malignancy (no evidence of disease in CSF by flow cytometry) CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
• Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
• Uncontrolled active hepatitis B or hepatitis C
• Active HIV infection (controlled HIV is permissible)
• Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
• Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
• Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
• Intolerance to the excipients of the CAR-T cell product
• Any immunosuppressive medication must be stopped ≥ 2 weeks prior to apheresis.
• Patient has taken one of the prohibited concomitant medications within the timeframe. ARM C: Kymriah for rRelapsed or rRefractory diffuse large B cell lymphoma
Inclusion Criteria:

• Age and Disease Status
• Adult patients (age ≥ 18 years)
• with relapsed or refractory (r/r) large B-cell lymphoma, including
• diffuse large B-cell lymphoma (DLBCL) not otherwise specified,
• high grade B-cell lymphoma
• and DLBCL arising from follicular lymphoma.
• Disease status:
• after two or more lines of systemic therapy or
• relapse after autologous HCT
• Performance Status
• ECOG performance status 0-2
• ALC >/=100/uL at screening (prior to apheresis)
• Organ Function
• Renal function defined as:
• A serum creatinine of ≤1.5 x ULN OR
• eGFR ≥ 50 mL/min/1.73 m^2
• Liver function defined as:
• ALT ≤ 5 times the ULN for age (unless due to disease)
• Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN
• Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
• Hemodynamically stable and LVEF ≥ 45% confirmed by echocardiogram or MUGA
• Adequate bone marrow reserve (unless marrow infiltrated by disease) defined as :
• Absolute neutrophil count (ANC) > 1.000/mm3 (only for NHL)
• Platelets ≥ 50.000/mm3 (transfusion support can be provided)
• Hemoglobin >8.0 mg/dl (transfusion support can be provided)
• Other Inclusion Criteria
• Life expectancy ≥12 weeks
• Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment.
• Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:

• Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
• Active CNS involvement by malignancy (no evidence of disease in CSF by flow cytometry) CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
• Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
• Uncontrolled active hepatitis B or hepatitis C
• Active or inactive HIV infection
• Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
• Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
• Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
• Intolerance to the excipients of the CAR-T cell product
• Any immunosuppressive medication must be stopped ≥ 2 weeks prior to apheresis.
• Patient has taken one of the prohibited concomitant medications within the timeframe ARM D: Tecartus (Brexucabtagene Autoleucel) for relapsed or refractory mantle cell lymphoma
Inclusion Criteria:

• Age and Disease Status * with relapsed or refractory (r/r) mantle cell lymphoma, including
• prior anthracycline or Bendamustine containing therapy
• prior Rituximab or other CD20 directed antibody (or inability to treat with CD20 MoAb)
• not a candidate or relapse after autologous HCT
• active disease at enrollment
• Performance Status *ECOG performance status 0-1
• Organ Function
• Renal function defined as:
• A serum creatinine of ≤1.5 x ULN OR
• eGFR ≥ 50 mL/min/1.73 m2
• Liver function defined as:
• ALT ≤ 5 times the ULN for age (unless due to disease)
• Bilirubin ≤ 2.0 mg/dl with the exception of patients with Gilbert syndrome; may be included if their total bilirubin is ≤ 3.0 x ULN and direct bilirubin ≤ 1.5 x ULN
• Must have a minimum level of pulmonary reserve defined as ≤ Grade 1 dyspnea and pulse oxygenation SpO2 > 91% on room air
• Hemodynamically stable and LVEF ≥ 45% confirmed by echocardiogram or MUGA
• Adequate bone marrow reserve (unless marrow infiltrated by disease) defined as:
• Absolute neutrophil count (ANC) > 1,000/mm^3 (only for NHL)
• Platelets ≥ 50,000/mm^3 (transfusion support can be provided)
• Hemoglobin >8.0 mg/dl (transfusion support can be provided) Other
Inclusion Criteria:

• Life expectancy ≥12 weeks
• Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment. See section 4.5 for definitions of child bearing potential and section 4.6 for definitions of adequate birth control.
• Written voluntary consent (adults) or parental/guardian consent (minors or adults with diminished capacity) prior to the performance of any research related tests or procedures.
Exclusion Criteria:

• Pregnant or breastfeeding - Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
• Active CNS involvement by malignancy (no evidence of disease in CSF by flow cytometry) CAR-T is not indicated for the treatment of patients with primary central nervous system lymphoma.
• Presence of Grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). All GVHD medication must be stopped 2 weeks prior to apheresis.
• Uncontrolled active hepatitis B or hepatitis C
• Active HIV infection
• Uncontrolled acute life threatening bacterial, viral or fungal infection (e.g. blood culture positive ≤ 72 hours prior to infusion)
• Unstable angina and/or myocardial infarction within 1 month prior to CAR-T infusion
• Investigational medicinal product within the last 7 days prior to apheresis or CAR-T infusion
• Intolerance to the excipients of the CAR-T cell product
• Any immunosuppressive medication must be stopped ≥ 2 weeks prior to apheresis (steroids must be stopped >72 hours prior to apheresis).
• Patient has taken one of the prohibited concomitant medications within the timeframe
Drug: KYMRIAH, Drug: YESCARTA, Drug: Fludarabine 30mg/m2 4 doses, Drug: Cyclophosphamide 500 mg/m2, 2 doses, Drug: Fludarabine 30mg/m2 3 doses, Drug: Cyclophosphamide 500 mg/m2, 3 doses, Drug: Fludarabine 25mg/m2 3 days, Drug: Cyclophosphamide 250 mg/m2, 3 days, Drug: Tecartus
Acute Lymphoblastic Leukemia, Large B-cell Lymphoma
ALL, CAR-T, CAR19-T, chimeric antigen receptor T cells, Clinics and Surgery Center (CSC)
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