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298 Study Matches

VX20-121-102: A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)

This is a Phase 3, randomized, double-blind, ELX/TEZ/IVA-controlled, parallel-group, multicenter study to evaluate the efficacy of VX-21/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in cystic fibrosis (CF) subjects who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

Joanne Billings
All
12 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT05033080
STUDY00013058
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Key
Inclusion Criteria:

• Heterozygous for F508del and a minimal function mutation (F/MF genotype)
• Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving ELX/TEZ/IVA therapy; FEV1 >=40% and <=80% for participants not currently receiving ELX/TEZ/IVA Key
Exclusion Criteria:

• History of solid organ or hematological transplantation
• Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
• Lung infection with organisms associated with a more rapid decline in pulmonary status
• Pregnant or breast-feeding females Other protocol defined Inclusion/Exclusion criteria may apply
Drug: VX-121/TEZ/D-IVA, Drug: ELX/TEZ/IVA, Drug: IVA, Drug: Placebo (matched to VX-121/TEZ/D-IVA), Drug: Placebo (matched to ELX/TEZ/IVA), Drug: Placebo (matched to IVA)
Cystic Fibrosis, Rare Diseases
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Transcatheter Aortic Valve Replacement to UNload the Left ventricle in patients with ADvanced heart failure: a randomized trial

The objective of this study is to determine the safety and efficacy of transcatheter aortic valve replacement (TAVR) via a transfemoral approach in HF patients with moderate AS as compared with OHFT. International, multi-center, randomized, open-label, clinical trial comparing the safety and efficacy of TAVR with the SAPIEN 3 or SAPIEN 3 Ultra THV and OHFT versus OHFT in HF patients, with moderate AS. All patients are followed from randomization until at least 1 year after randomization of the last patient. Patients are followed from randomization until 1 year after randomization for the last patient (efficacy assessment time interval [EATI]).

Mudassar Ahmed
Pivotal
This study is NOT accepting healthy volunteers
NCT02661451
STUDY00009416
Aortic Valve Stenosis
Valve
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A Phase 2 Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients with Chronic Inflammatory Demyelinating Polyneuropathy

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare neurological disorder in which there is inflammation of nerve roots and peripheral nerves and destruction of the fatty protective covering (myelin sheath) over the nerves. There is an unmet medical need for an efficacious treatment with a favorable safety and tolerability profile and more convenient administration than that provided by current treatments. A weekly subcutaneous (SC) treatment option consisting of efgartigimod PH20 SC administered within a few minutes could offer clinically significant benefits to CIDP patients. Stage A (open-label, efgartigimod PH20 SC; 4-12 weeks) Primary objective: to assess the activity of efgartigimod PH20 SC based on the percentage of patients classified as treatment responders. Stage B (double-blind, randomized-withdrawal, efgartigimod PH20 SC or placebo; up to 48 weeks) Primary objective: to determine the efficacy of efgartigimod PH20 SC compared to placebo based on the time needed for the occurrence of the first evidence of clinical deterioration.

Jeff Allen
Phase II
This study is NOT accepting healthy volunteers
NCT04281472
STUDY00008212
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Rare Diseases
Clinics and Surgery Center (CSC)
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Influence of Cooling duration on Efficacy in Cardiac Arrest Patients

A multicenter, randomized, adaptive allocation clinical trial to determine if increasing durations of induced hypothermia are associated with an increasing rate of good neurological outcomes and to identify the optimal duration of induced hypothermia for neuroprotection in comatose survivors of cardiac arrest.

Benjamin Miller
Phase II/III
This study is NOT accepting healthy volunteers
NCT04217551
STUDY00011864
Cardiac Arrest, Out-Of-Hospital, Hypothermia, Induced, Hypoxia-Ischemia, Brain
Bayesian Adaptive Clinical Trial, Coma, Hypothermia, therapeutic
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MT2020-39: AGAVE-201, A Phase 2, Open-label, Randomized, Multicenter, Study to Evaluate the Efficacy, Safety, and Tolerability of Axatilimab at 3 Different Doses in Patients with Recurrent of Refractory Active Chronic Graft Versus Host Disease who have Received at least 2 Lines of Systemic Therapy (AGAVE-201)

The purpose of this study is to compare how well axatilimab helps cGVHD at different dose levels and to find out whether there are side effects from its use.

Shernan Holtan
All
2 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04710576
STUDY00011666
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Inclusion Criteria:

• Participants must be 2 years of age or older, at the time of signing the informed consent.
• Participants who are allogeneic hematopoietic stem cell transplantation (HSCT) recipients with active cGVHD requiring systemic immune suppression. Active cGVHD is defined as the presence of signs and symptoms of cGVHD per 2014 NIH Consensus Development Project on Criteria for Clinical trials in cGVHD.
• Participants with refractory or recurrent active cGVHD despite at least 2 lines of systemic therapy.
• Refractory disease defined as meeting any of the following criteria:
• The development of 1 or more new sites of disease while being treated for cGVHD.
• Progression of existing sites of disease despite at least 1 month of standard or investigation therapy for cGVHD.
• Participants who have not achieved a response within 3 months on their prior therapy for cGVHD and for whom the treating physician believes a new systemic therapy is required.
• Recurrent cGVHD is active, symptomatic disease (after an initial response to prior therapy) as defined, based on the NIH 2014 consensus criteria, by organ-specific or global assessment or for which the physician believes that a new line of systemic therapy is required.
• Participants may have persistent, active acute and cGVHD manifestations (overlap syndrome), as defined by 2014 NIH Consensus Development Project on Criteria for Clinical trials in cGVHD.
• Karnofsky Performance Scale of ≥60 (if aged 16 years or older); Lansky Performance Score of ≥60 (if aged <16 years)
• Adequate organ and bone marrow functions evaluated during the 14 days prior to randomization.
• Creatinine clearance (CrCl) ≥30 milliliter/minute/1.73 square meter based on the Cockcroft-Gault formula in adult participants and Schwartz formula in pediatric participants.
• Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• Concomitant use a of systemic corticosteroid is allowed but not required. Topical and inhaled corticosteroid agents are allowed. If a participant is taking corticosteroids at study randomization, they must be on a stable dose of corticosteroids for at least 2 weeks prior to Cycle 1 Day 1.
• Concomitant use of CNI or sirolimus is allowed but not required.
• Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and protocol. A parent/guardian should provide consent for pediatric participants unable to provide consent themselves; in addition, where applicable pediatric participants should sign their own assent form.
Exclusion Criteria:
Participants are excluded from the study if any of the following criteria apply:
• Has acute GVHD without manifestations of cGVHD.
• Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening.
• History of acute or chronic pancreatitis.
• History of myositis.
• History or other evidence of severe illness, uncontrolled infection or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study.
• Participants with acquired immune deficiency syndrome (AIDS).
• Hepatitis B (defined as hepatitis B virus [HBV] surface antigen positive and HBV core antibody positive, with positive HBV deoxyribonucleic acid [DNA], or HBV positive core antibody alone with positive HBV DNA. Hepatitis C (defined as positive hepatitis C [HCV] antibody with positive HCV ribonucleic acid [RNA]).
• Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years of randomization, unless previously treated with curative intent and approved by Sponsor's Medical Monitor (for example, completely resected basal cell or squamous cell carcinoma of the skin, resected in situ cervical malignancy, resected breast ductal carcinoma in situ, or low-risk prostate cancer after curative resection).
• Female participant who is pregnant or breastfeeding.
• Previous exposure to CSF1-R targeted therapies.
• Taking agents for treatment of cGVHD other than corticosteroids and either a CNI or sirolimus is prohibited.
• For approved or commonly used agents, other than corticosteroids, CNI and sirolimus, a washout of 2 weeks or 5 half-lives, whichever is shorter, is required at study enrollment.
• Receiving another investigational treatment within 28 days of randomization.
• Participants should not be participating in any other interventional study. Pediatric participants are encouraged to also participate in the ongoing developmental studies of the Pediatric cGVHD Symptom Scale (PCSS).
Drug: Axatilimab
Chronic Graft-versus-host-disease
cGVHD, AGAVE-201, GVHD, graft versus host disease, graft-versus-host-disease, Clinics and Surgery Center (CSC)
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Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry

The primary objective for this observational study is to collect general and medical data from children, adolescents, and young adults who had pediatric onset rheumatic disease. This data will be used to evaluate the long-term safety and efficacy of therapeutic agents used to treat these diseases. This information will allow investigators to accurately report and follow changes in current medication use patterns and compare these to proposed standards and current treatment recommendations. The use of a single registry will allow for more analysis of the different therapeutic agents by allowing them to be compared to each other.

Colleen Correll
All
up to 21 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02418442
1506M74443
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Inclusion Criteria:

• Onset of rheumatic disease prior to age 16 years for JIA and onset prior to age 19 years for all other rheumatic diseases (see appendix A).
• Subject (and/or parent/legal guardian when required) is able to provide written informed consent and willing to comply with study procedures.
• Subject and/or parent/legal guardian is willing to be contacted in the future by study staff.
Exclusion Criteria:

• Greater than 21 years of age at the time of enrollment.
Rheumatic Joint Disease
Systemic Arthritis, Oligoarthritis, Polyarthritis (Rheumatoid Factor Negative), Polyarthritis (Rheumatoid Factor Positive), Psoriatic Arthritis, Enthesitis Related Arthritis (ERA), Undifferianted Arthritis, CARRA Consensus Treatment Plans
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MultiStem Administration for Stroke Treatment and Enhanced Recovery Study

This study is evaluating the efficacy of MultiStem (drug) on functional outcome in participants with ischemic stroke.

Muhammad Affan
Phase III
This study is NOT accepting healthy volunteers
NCT03545607
STUDY00008000
Ischemic Stroke
adult stem cells, ischemic stroke
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A Randomized, Double-Blind, Multicenter, Placebo-Controlled Phase 3 Study with Open-Label Period to Evaluate the Efficacy and Safety of Inebilizumab in Adults with Myasthenia Gravis

This is a randomized, double-blind, placebo-controlled, parallel-group study with an optional open-label extension testing the safety and efficacy of inebilizumab in participants with Myasthenia Gravis.

Georgios Manousakis
Phase III
This study is NOT accepting healthy volunteers
NCT04524273
STUDY00010270
Myasthenia Gravis, Rare Diseases
Clinics and Surgery Center (CSC), Myasthenia Gravis
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Long-Term Follow-up Protocol for Participants Treated With Gene-Modified T Cells

This study is designed for the following purpose: - To assess the risk of delayed adverse events (AEs) following exposure to genemodified(GM) T cells - To monitor for long-term persistence of GM T cells, including analysis of vector integration sites, as appropriate. - To monitor for generation of replication competent retroviruses (RCR) - To assess long-term efficacy following treatment with GM T cells - Describe growth, developmental outcome, and sexual maturity status for subjects who were aged < 18 years at time of GM T cell treatment - To assess long term health-related quality of life following treatment with GM T cells

Joseph Maakaron
All
Not specified
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT03435796
STUDY00010571
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Inclusion Criteria:

• Received at least one gene-modified (GM) T-cell infusion in a previous Celgene sponsored or Celgene alliance partner-sponsored study, and have discontinued, or completed the post-treatment follow-up period in the parent treatment protocol, as applicable.
• Must understand and voluntarily sign an Informed Consent Form/Informed Assent Form prior to any study-related assessments/procedures being conducted.
Exclusion Criteria:
Not Applicable Other protocol-defined inclusion/exclusion criteria apply
Genetic: Gene-modified (GM) T cell therapy
Neoplasms
Long-term follow up, Gene-Modified T Cells, CAR T Cell
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A natural history study to TRACK brain and spinal changes in individuals with Friedreich Ataxia (TRACK-FA) ((TRACK-FA))

TRACK-FA is a multi-site, international, prospective, observational neuroimaging study in Friedreich’s Ataxia. The objective is to obtain natural history MRI and MRS data over time in the brain and spinal cord in a large cohort of FRDA patients and healthy control. This will permit validation of neuroimaging biomarkers in FRDA for future clinical trials. In addition to neuroimaging data, the study will collect data from clinical, cognitive and mood assessments as well as blood samples to measure blood markers (frataxin and neurofilament light chain). The present IRB protocol covers only subjects enrolled at the University of Minnesota.

Pierre-Gilles Henry
All
5 Years and over
NA
This study is NOT accepting healthy volunteers
NCT04349514
STUDY00009047
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Inclusion Criteria:

• Age ≥ 5 years
• Written informed consent provided
• Individuals with FA must have a genetic confirmation of diagnosis and be biallelic for a GAA repeat length > 55 in intron 1 of FXN and/or have a GAA repeat length > 55 in intron 1 of FXN in one allele and another type of mutation that is inferred to cause loss of function in the second FXN allele
• Individuals with FA must have an age of disease onset ≤ 25 years
• Individuals with FA must have a disease duration ≤ 25 years
• Individuals with FA must have a Friedreich Ataxia Rating Scale (FARS) Functional staging score of ≤ 5 and total modified FARS (mFARS) score of ≤ 65 on enrolment
Exclusion Criteria:

• Age < 5 years
• Unable to provide written informed consent
• Magnetic resonance contraindications (e.g. pacemaker or other metallic surgical implants)
• Presence of metallic dental braces
• Pregnancy (ascertained via a question or test as mandated at particular sites)
• Individuals with FA must not have acute or ongoing medical or other conditions that, after discussion between the Site Investigator and steering committee, is deemed to interfere with the conduct and assessments of the study
• Individuals with FA must not have another neurological condition apart from FA
• Individuals with FA must not have other neurologic conditions that, in the opinion of the Site Investigator, would interfere with the conduct and assessments of the study
• Controls must not have a diagnosed psychiatric or neurological condition
• Controls must not have acute or ongoing medical or other conditions that would interfere with the conduct and assessments of the study
Other: Natural history
Friedreich Ataxia
Neuroimaging, Longitudinal, Biomarkers, Natural history
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MT2013-06C : Treatment of graft Failure after HSCT

Troy Lund
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT02161783
1404M49341
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Inclusion Criteria:

• Patients with primary or secondary graft failure, as defined below, may receive a second transplant:
• Primary graft failure is defined as not achieving an ANC ≥0.5x10^9/L for three consecutive days by day 35 - 42 following the first transplant.
• Secondary graft failure is defined as achieving an ANC ≥0.5x10^9/L for three consecutive days by day 35 - 42, but subsequently drops below 0.5x10^9/L without recovery.
• Loss of chimerism is defined as achieving an ANC ≥0.5x10^9/L for three consecutive, but with less than 10% CD15+ donor cells in the marrow or peripheral blood.
• Recipients should have acceptable organ function defined as:
• Renal: creatinine < 2.0 (adults) and creatinine clearance > 30. For creatinine clearance < 70, consultation with a BMT pharmacist is necessary for chemotherapy dose adjustments.
• Hepatic: bilirubin, AST/ALT, ALP < 10 x upper limit of normal
• Cardiac: left ventricular ejection fraction > 40%
Exclusion Criteria:

• Uncontrolled infection at the time of transplant.
• Patients with Fanconi Anemia or other DNA breakage syndromes.
Drug: Fludarabine, Drug: Cyclophosphamide, Radiation: Total Body Irradiation, Biological: Hematopoietic stem cell infusion
Primary Graft Failure, Secondary Graft Failure
Hematopoietic stem cell transplant, Clinics and Surgery Center (CSC)
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A RANDOMIZED PHASE III STUDY OF IBRUTINIB PLUS OBINUTUZUMAB VERSUS IBRUTINIB PLUS VENETOCLAX AND OBINUTUZUMAB IN UNTREATED OLDER PATIENTS (&#8805; 70 YEARS OF AGE) WITH CHRONIC LYMPHOCYTIC LEUKEMIA (CLL)

Birendra Kumar
Phase III
This study is NOT accepting healthy volunteers
NCT03737981
0123456789
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
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A Phase 2/3, Two-Part, Open-Label, Dose Escalation, Age De-escalation and Randomized, Observer-Blind, Placebo-Controlled Expansion Study to Evaluate the Safety, Tolerability, Reactogenicity, and Effectiveness of mRNA-1273 SARS-CoV-2 Vaccine in Healthy Children 6 months to < 12 Years of Age (mRNA-1273-P204) - COVID-19

The Sponsor of this study, ModernaTX, is studying the mRNA-1273 vaccine for the prevention of COVID-19 in children. This study is being conducted to learn about the safety, any side effects, and how your child’s body responds to the study vaccine (the “immune response”).

Shane McAllister
Phase II/III
This study is also accepting healthy volunteers
NCT04796896
STUDY00012613
SARS-CoV-2
COVID-19, COVID-19 Vaccine, Coronavirus, Messenger RNA, Moderna, SARS-CoV-2, SARS-CoV-2 Vaccine, Virus Diseases, mRNA-1273, mRNA-1273 vaccine
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MT2021-11: An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects with Epstein-Barr Virus-associated Diseases

This study is intended to determine the clinical benefit of tabelecleucel (EBV-specific cytotoxic T-lymphocytes) in subjects with EBV-associated diseases.

Joseph Maakaron
All
Not specified
Phase 2
This study is NOT accepting healthy volunteers
NCT04554914
STUDY00013494
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Inclusion Criteria:

• Diagnosis of EBV+ disorder
• Eastern Cooperative Oncology Group performance status <= 3 for participants aged >= 16 years; Lansky score >= 20 for participants from 1 year to < 16 years
• Adequate organ function test results, unless organ dysfunction is considered to be due to the underlying EBV-associated disease by the investigator Cohort-specific
Inclusion Criteria:

• For participants with PID LPD:
• Newly diagnosed or relapsed/refractory LPD confirmed by biopsy-proven EBV+ LPD or positive cerebrospinal fluid (CSF) cytology with or without radiographically measurable intracranial disease with EBV detected in CSF
• Participant must have systemic measurable disease and/ or CNS measurable disease
• Definitive therapy (eg, allogeneic HCT, gene therapy) for the underlying PID is planned
• Participants with newly diagnosed disease should be ineligible for standard first-line therapy for EBV+ LPD, as determined by the investigator
• For participants with AID LPD:
• Newly diagnosed or relapsed/refractory LPD confirmed by biopsy-proven EBV+ LPD or positive CSF cytology, with or without radiographically measurable intracranial disease, with EBV detected in CSF
• Participant must have systemic measurable disease and/ or CNS measurable disease
• Participants who are human immunodeficiency virus positive (HIV+) must meet both of the following criteria: Have an HIV viral load assessed by reverse transcription-polymerase chain reaction (RT-PCR) below the lower limit of detection and CD4 >= 50 cells/μL within 6 months prior to the first dose of tabelecleucel
• Participants with newly diagnosed disease should be ineligible for standard first-line therapy for EBV+ LPD, as determined by the investigator
• For participants with CNS PTLD:
• Newly diagnosed or relapsed/refractory EBV+ CNS PTLD histologically confirmed by biopsy-proven EBV+ CNS PTLD or positive CSF cytology with or without radiographically measurable intracranial disease with EBV detected in CSF
• Participant may have systemic and CNS disease or CNS disease only
• Participants with newly diagnosed disease should be ineligible for standard first-line therapy for EBV+ LPD, as determined by the investigator
• For participants with EBV+ PTLD, where standard first line therapy (rituximab and/or chemotherapy) is not appropriate, including CD20-negative disease:
• Newly diagnosed, biopsy-proven EBV+ PTLD
• Ineligible for standard first-line therapy for EBV+ PTLD, as determined by the investigator
• Participants must have systemic disease measurable per Lugano Classification criteria, except when contraindicated or mandated by local practice, then MRI may be used.
• For participants with sarcoma, including LMS:
• Newly diagnosed or failed systemic first-line therapy for EBV+ sarcoma. Participants with newly diagnosed disease should be ineligible for standard first-line therapy for EBV+ sarcoma, as determined by the investigator.
• Biopsy-proven EBV+ sarcoma
• Measurable disease using diagnostic PET/CT and/or MRI following RECIST 1.1 criteria
• For participants with CAEBV:
• Newly diagnosed or previously treated CAEBV
• Detectable EBV viremia on at least 2 occasions at a minimum of 90 days apart
• At least 3 active clinical findings (per Kimura H, et al. Front Immunol. 2017;8:1867) as: Fever >= 38.5°C; splenomegaly, lymphadenopathy, and/or hepatomegaly; cytopenia affecting at least 2 or 3 lineages in the peripheral blood (hemoglobin < 9 g/dL, platelets < 100 × 10^3/mL, neutrophils < 1 × 10^3/mL); hypogammaglobulinemia; hemophagocytosis; hepatitis; neuropathy; rash; and hydroa vacciniforme
• For participants with EBV+ viremia with HLH:
• Newly diagnosed or previously treated EBV+ viremia with HLH
• A molecular diagnosis consistent with HLH-2004 trial (per Henter JI, et al. Pediatr Blood Cancer. 2007;48:124-31) OR 5 or more of the clinical symptoms (per Jordan MB, et al. Blood. 2011;118:4041-4052): Fever >= 38.5°C; splenomegaly; cytopenia affecting at least 2 or 3 lineages in the peripheral blood (hemoglobin < 9 g/dL, platelets < 100 × 10^3/mL, neutrophils < 1 × 10^3/mL); hypertriglyceridemia (fasting >= 265 mg/dL) and/or hypofibrinogenemia (<= 150 mg/dL); hemophagocytosis in bone marrow, spleen, lymph nodes, or liver; low or absent natural killer cell (NK-cell) activity; ferritin >= 500 ng/mL; and elevated soluble CD25
Exclusion Criteria:

• Burkitt, T-cell (except in the setting of HLH), natural killer/T-cell lymphoma/LPD, Hodgkin, or transformed lymphoma
• Serious known active infections, defined as ongoing uncontrolled adenovirus infection or infections requiring systemic therapy at the time of enrollment
• Suspected or confirmed Grade >= 2 acute graft-versus-host disease (GvHD) per the Center for International Blood and Marrow Transplant Research (CIBMTR) consensus grading system or extensive chronic GvHD per National Institutes of Health (NIH) consensus criteria at the time of the enrollment
• Need for vasopressor or ventilatory support
• Prior therapy (in order of increasing washout period) prior to enrollment as:
• Within 4 weeks or 5 half-lives (whichever is shorter) for any investigational product and/ or any chemotherapy (systemic or intrathecal), targeted small molecule therapy, or antibody/biologic therapy. Note: prior anti-CD20 antibody use is permitted within the washout period if a subsequent disease response assessment indicates disease progression
• Within <= 8 weeks for cellular therapies (EBV-CTLs, chimeric antigen receptor therapies directed at T cells or T-cell subsets, donor lymphocyte infusion, other CTLs); and/or therapies which could impact tabelecleucel function (anti-thymocyte globulin, alemtuzumab)
• Unwilling to use protocol specified contraceptive methods
• Women who are pregnant or breastfeeding
• Ongoing need for daily steroids of > 0.5 mg/kg prednisone or glucocorticoid equivalent, ongoing methotrexate, or extracorporeal photopheresis (protocol-specified dexamethasone is permitted and concludes by the time of enrollment)
• For participants with PID LPD or AID LPD: history of prior allogeneic HCT or solid organ transplant
Biological: Tabelecleucel
Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (PID LPD), EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (AID LPD), EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (CNS PTLD), EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD), Solid Organ Transplant Complications, Lymphoproliferative Disorders, Allogeneic Hematopoietic Cell Transplant, Stem Cell Transplant Complications, EBV+ Sarcomas, Leiomyosarcoma, Chronic Active Epstein-Barr Virus (CAEBV), Chronic Active Epstein-Barr Virus With Hemophagocytic Lymphohistiocytosis (HLH), Lymphohistiocytosis, Hemophagocytic
Allogeneic, Off-The-Shelf T-cell Immunotherapy, Epstein-Barr Virus (EBV), Epstein-Barr Virus-specific Cytotoxic T lymphocyte (EBV-CTL), Solid Organ Transplant (SOT), Hematopoietic Cell Transplant (HCT), Clinics and Surgery Center (CSC)
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MT2012-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Christen Ebens
All
up to 50 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01652092
1207M17321
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Inclusion Criteria:

• Diagnosis of immunodeficiency or histiocytic disorder including the following:
• Severe combined immunodeficiency (SCID - all variants)
• Second bone marrow transplant (BMT) for SCID (after graft rejection)
• Omenn's Syndrome
• Reticular dysgenesis
• Wiskott-Aldrich syndrome
• Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)
• Hyper IgM Syndrome (CD40 Ligand Deficiency)
• Common variable immunodeficiency (CVID) with severe phenotype
• Chronic Granulomatous Disease (CGD)
• Other severe Combined Immune Deficiencies (CID)
• Hemophagocytic Lymphohistiocytosis (HLH)
• X-linked Lymphoproliferative Disease (XLP)
• Chediak-Higashi Syndrome (CHS)
• Griscelli Syndrome
• Langerhans Cell Histiocytosis (LCH)
• Acceptable stem cell sources include:
• HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow
• HLA identical or up to a 1 antigen mismatched unrelated BM donor
• Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards
• Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of >5 x 10^7 nucleated cells/kg as per current institutional guidelines
• Double unrelated umbilical cord blood units that are:
• up to 2 antigen mismatched to the patient
• up to 2 antigen mismatched to each other
• minimum cell dose of at least one single unit must be ≥ 3.5 x 10^7 nucleated cells/kg
• combined dose of both units must provide a total cell dose of ≥ 5 x 10^7 nucleated cells/kg
• Age: 0 to 50 years
• Adequate organ function and performance status. Exclusion Criteria
• pregnant or breastfeeding
• active, uncontrolled infection and/or HIV positive
• acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
Drug: Alemtuzumab 0.3 mg, Drug: Cyclophosphamide, Drug: Busulfan, Biological: Stem Cell Transplantation, Drug: Fludarabine phosphate 40 mg, Drug: Melphalan, Drug: Alemtuzumab 0.2 mg, Drug: Busulfan, Drug: Fludarabine phosphate 30 mg, Drug: MESNA
SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis
immunodeficiency disorder, histiocytic disorder, Clinics and Surgery Center (CSC)
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A Randomized Phase III Study of the addition of Venetoclax to Ibrutinib and Obinutuzumab versus Ibrutinib and Obinutuzumab in Untreated Younger Patients with Chronic Lymphocytic Leukemia (CLL)

Birendra Kumar
Phase III
This study is NOT accepting healthy volunteers
NCT03701282
0123456789
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
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MT2017-28 The Head Start 4 Protocol - Newly Diagnosed Children (less than 10 years old) with Medulloblastoma and Other Central Nervous System Embryonal Tumors: Clinical and Molecular Risk-Tailored Intensive and Compressed Induction Chemotherapy Followed by Consolidation with Either Single Cycle (Low Risk Patients) or Randomization (High Risk Patients) to Either Single-Cycle or to Three Tandem Cycles of Marrow-Ablative Chemotherapy with Autologous Hematopoietic Progenitor Cell Rescue Added Title: Neuroanatomical, Cognitive and Family Aspects to Recovery from a Brain Tumor

This is a prospective randomized clinical trial, to determine whether dose-intensive tandem Consolidation, in a randomized comparison with single cycle Consolidation, provides an event-free survival (EFS) and overall survival (OS). The study population (children < 120 months) will be high-risk patients (non-Wnt and non-Shh sub-groups) with medulloblastoma, and for all patients with central nervous system (CNS) embryonal tumors completing "Head Start 4" Induction. This study will further determine whether the additional labor intensity (duration of hospitalizations and short-term and long-term morbidities) associated with the tandem treatment is justified by the improvement in outcome. It is expected that the tandem (3 cycles) Consolidation regimen will produce a superior outcome compared to the single cycle Consolidation, given the substantially higher dose intensity of the tandem regimen, without significant addition of either short-term or long-term morbidities.

Christopher Moertel, MD
All
up to 10 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT02875314
STUDY00000427
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Inclusion Criteria:

• Patients 10 years of age at the time of definitive confirmatory eligible histologic or cytologic diagnosis of eligible CNS tumor (brain or spinal cord)
• Patients may not have received irradiation or chemotherapy (except corticosteroids)
• Have histologically proven diagnosis of medulloblastoma or CNS embryonal tumors of the brain or spinal cord
• Medulloblastoma
• Posterior fossa classic, desmoplastic or extensive nodular or anaplastic/large cell medulloblastoma with appropriate and sufficient tumor material (FFPE or snap frozen) for proposed assays: all stages, age less than 6 years at diagnosis
• Posterior fossa classic or anaplastic/large cell medulloblastoma with sufficient tumor material (FFPE or snap frozen) for proposed assays: clinically high-stage (neuraxis or extra-neural dissemination, M1-4), age greater than 6 years to less than 10 years at diagnosis
• Posterior fossa medulloblastoma, those 6 years of age and above at diagnosis, will only be eligible if they have evidence of neuraxis or extraneural dissemination. Patients 6 years of age and above with low-stage (standard-risk, M0) medulloblastoma will NOT be eligible for this study, irrespective of molecular subgroup and extend of local resection
• CNS Embryonal Tumors:
• Pineoblastoma, CNS neuroblastoma, CNS ganglioneuroblastoma, embryonal tumor with multi-layered rosettes (ETMR, including embryonal tumor with abundant neuropil and true rosettes (ETANTR), ependymoblastoma and ETMR not otherwise specified), medulloepithelioma, CNS embryonal tumor with rhabdoid features (INI1 intact) and CNS embryonal tumor, not otherwise specified.
• Must commence Induction chemotherapy within 28 days of the most recent definitive surgical procedure and within 21 days of the most recent neuro-imaging studies (MRI of brain, performed with and without gadolinium contrast, and MRI of total spine, performed with gadolinium contrast) and lumbar CSF cytological examination
• Patients must have adequate organ functions at the time of registration:
• Liver: bilirubin less than 1.5 mg/dL (except for patients with Gilbert's Syndrome of indirect hyperbilirubinemia) and transaminases [SGPT or ALT, and SGOT or AST] less than 2.5 (two and a half) times the upper limits of institutional normal.
• Renal: Creatinine clearance and/or glomerular filtration rate (GFR) greater than or equal to 60 mL/min/1.73m² within 21 days of protocol therapy.
• Bone Marrow Function:
• Peripheral absolute phagocyte count (APC) > 1000/ µL. APC = numbers of banded neutrophils + segmented neutrophils + metamyelocytes + monocytes + eosinophils Please note, if institution reports differential as a percentage, then APC = [percentage of banded neutrophils + segmented neutrophils+ metamyelocytes+monocytes+eosinophils] x total white cell count.
• Platelet Count > 100,000/µL (transfusion independent)
• Hemoglobin > 8 gm/dL (may have received RBC transfusions).
Exclusion Criteria:

• Patients older than 10 years of age at time of diagnosis
• Following diagnoses are not eligible for study enrollment: CNS atypical teratoid/rhabdoid tumor (AT/RT); all ependymomas including anaplastic ependymomas of the brain or spinal cord; all choroid plexus carcinomas; all high-grade glial and glio-neuronal tumors; all primary CNS germ cell tumors; all primary CNS sarcomas; all primary or metastatic CNS lymphomas and solid leukemic lesions (i.e., chloromas, granulocytic sarcomas).
• Patients with unbiopsied diffuse intrinsic pontine tumors will NOT be eligible for this study.
Drug: Induction, Drug: Single Cycle Intensive Chemotherapy, Drug: Tandem 3 Cycle Intensive Chemotherapy
Medulloblastoma, Central Nervous System Embryonal Tumors
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A STUDY EVALUATING THE PREGNANCY OUTCOMES AND SAFETY OF INTERRUPTING ENDOCRINE THERAPY FOR YOUNG WOMEN WITH ENDOCRINE RESPONSIVE BREAST CANCER WHO DESIRE PREGNANCY (POSITIVE)

NA
This study is NOT accepting healthy volunteers
NCT02308085
0123456789
Early Breast Cancer
Breast, Pregnancy, Premenopausal, endocrine responsive
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Development of Ultra-Low Dose CT Based Screening for Aortic Aneurysm

The primary objective of this research project is development and validation of a new, non-contrast gated aortic (NCGA) computer tomography scan algorithm for screening of aortic aneurysm in the chest and abdomen in at risk patients. This study would initially be performed in patients with a known aneurysm and done in addition to their indicated surveillance CT scan.

Rumi Faizer
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT03479164
1510M79442
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Inclusion Criteria:

• Adult patients who carry the diagnosis of thoracic aortic aneurysm, aortic dissection, or abdominal aortic aneurysm and require CT imaging to evaluate the pathology
Exclusion Criteria:

• Current pregnancy
Other: Ultra Low-Dose CT
Aortic Aneurysm, Thoracic, Aortic Aneurysm, Abdominal
Clinics and Surgery Center (CSC)
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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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Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas (POPLAR-NF2)

This study will investigate the efficacy and safety of REC-2282 in patients with progressive NF2 mutated meningiomas who have either NF2 disease-related meningioma or recurrent sporadic meningiomas that have NF2 mutations. This study is a parallel-group, two-staged, Phase 2/3, randomized, multi-center study with two cohorts: Cohort A followed by Cohort B. The purpose of Cohort A is to provide early data on efficacy and safety of REC-2282 in participants with progressive NF2 mutated meningiomas, and provide guidance for the correct dose, population, sample size, and endpoint for the confirmatory part of the study (Cohort B). Additional goals for Cohort A are to assess effects of food on drug absorption. The purpose of Cohort B of the study is to assess the efficacy and safety of REC-2282 compared with placebo in participants with progressive NF2 mutated meningiomas.

All
12 Years and over
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT05130866
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Inclusion Criteria:

• ≥12 years of age and weighing at least 40 kg
• Progressive meningioma that is amenable to volumetric analysis
• Has either 1) sporadic meningioma with confirmed NF2 mutation; or, 2) confirmed diagnosis of NF2 disease (revised Manchester criteria); or, 3) at least one NF2-related tumor (with pathogenic germline or proven mosaic NF2 variant)
• Adequate bone marrow function
• Has provided written informed consent/assent to participate in the study
Exclusion Criteria:

• Progressive disease associated with significant or disabling clinical symptoms likely to require surgery or radiation therapy within the next 3 months.
• Received prior surgery, radiosurgery, or laser interstitial thermal therapy in the target tumor, or immediately adjacent to the target tumor within 6 months prior to screening.
• Received an anti- tumor agent for meningioma within 3 months, or 5 half-lives (whichever is longer), prior to screening.
• History of an active malignancy within the previous 3 years except for localized cancers that are considered cured, and, in the opinion of the investigator, present a low risk of recurrence.
• Received another investigational drug within 30 days prior to screening
• Pregnant, lactating, or is planning to attempt to become pregnant or impregnate someone during this study or within 90 days after the last dose of IMP.
Drug: REC-2282, Drug: Placebo
Neurofibromatosis Type 2
Neurofibromatosis Type 2, Neurofibromatosis Type II
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Adaptive Interventions for Smoking Cessation in Lung Cancer Screening Programs

In this sequential, multiple assignment, randomized trial (SMART) current smokers who are eligible for lung cancer screening will be identified using the electronic medical record at the University of Minnesota, Minneapolis VA, and Allina Health (N=1000). All participants will receive 8 weeks of evidence-based first-line smoking cessation treatment. Participants will be eligible for three potential randomizations during one year of smoking intervention: 1) to timing of identifying early response to treatment at 4 vs. 8 weeks (all participants), 2) to telephone-based tobacco longitudinal care (TLC) vs. TLC plus pharmacist-administered Medication Therapy Management (incomplete responders to first-line treatment, Primary Aim), and 3) to monthly TLC contact vs. quarterly TLC contact (complete responders to first-line treatment, Secondary Aim). The primary outcome will be 6 months of prolonged abstinence measured 18 months after the beginning of treatment.

Anne Joseph
NA
This study is NOT accepting healthy volunteers
NCT02597491
1506M74221
Smoking
Lung cancer screening, Sequential, multiple assignment, randomized trials (SMART), Smoking cessation, Smoking,
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STRIKE-PE: A Prospective, Multicenter Study of the IndigoTM Aspiration System Seeking to Evaluate the Long-Term Safety and Outcomes of Treating Pulmonary Embolism

The purpose of this study is to collect information on how patients with PE recover after treatment with the Indigo Aspiration System. The Indigo Aspiration System is a medical device that has been cleared by the U.S. Food and Drug Administration (FDA) for removing blood clots from the blood vessels throughout the body, excluding the head. The device is commercially available globally. Participants will be in this research study for about one year. Participants will be asked to complete a screening and baseline visit, device procedure in-patient visit as part of routine treatment for their PE, one post-procedure visit in the hospital and two follow-up visits. The study team will collect information on tests and procedures done during these visits from their medical records. They will also be asked to complete a quality of life questionnaire.

Michael Rosenberg
Post Market Monitoring
This study is NOT accepting healthy volunteers
NCT04798261
STUDY00013412
Pulmonary Embolism
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MT2016-15 :Reduced Intensity (RIC) Conditioning And Transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT) For Patients With Hematologic Malignancies

This is a two stage phase II trial of HLA-haploidentical related hematopoietic cells transplant (Haplo-HCT) using a reduced intensity conditioning (cyclophosphamide, fludarabine, melphalan, total body irradiation) with modifications based on factors including age and comorbidities. Bone marrow is the donor graft source. This study is designed to estimate disease-free survival (DFS) at 1 year posttransplant. Since the goal is to estimate the DFS at a long-term time-point (1 year post-transplant), the design is a generalization of the Simon design. Rather than suspension of the trial for evaluation after stage 1, this design uses an optimal interim analysis for futility without suspension of accrual.

Najla El Jurdi
All
up to 75 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02988466
1610M96901
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Inclusion Criteria:

• Karnofsky performance status of ≥70% or Lansky play score ≥ 70%
• A related haploidentical bone marrow donor with up to 2 or 3 HLA locus-mismatches
• The donor and recipient must be HLA identical for at least one haplotype (using high resolution DNA based typing) at the following genetic loci: HLA-A, HLA-B, HLA-C, and HLA-DRB1.
• Adequate liver and renal function
• Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction ≥ 40%
• Diffusion capacity corrected (DLCOcorr) > 40% predicted, and absence of O2 requirements
• > 6 months after prior autologous transplant (if applicable)
• Agrees to use contraception during study treatment
• Voluntary written consent (adult or parent/guardian with presentation of the minor information sheet, if appropriate)
• Patients who are HIV+ must have undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation
Exclusion Criteria:

• < 70 years with an available 5-6/6 HLA-A, B, DRB1 matched sibling donor
• Pregnancy or breastfeeding
• Current active and uncontrolled serious infection
• Acute leukemia in morphologic relapse/persistent disease defined as > 5% blasts in normocellular bone marrow OR any % blasts if blasts have unique morphologic markers (e.g. Auer rods).
• CML in blast crisis
• Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressive on salvage therapy.
• stable non-bulky disease is acceptable.
• Active central nervous system malignancy Criteria For Donor Selection:
• Donors must be HLA-haploidentical relatives of the patient, defined as having a shared HLA haplotype between donor and patient at HLA-A, -B, -C, and -DRB1.
• Eligible donors (14-70 years old) include biological children, siblings or half siblings, or parents, able and willing to undergo bone marrow harvesting.
• For donors <18 years, the maximum recipient weight (actual body weight) should not exceed 1.25 times the donor weight (actual body weight)1 In addition, bone marrow product volume should be limited to 20 ml/kg donor weight for donors <18 years.
Biological: Haplo HCT <55 years old, Biological: Haplo HCT ≥55 years old, Drug: GVHD Prophylaxis, Biological: Haplo HCT ≥55 and < 65 years old, Biological: Haplo HCT ≥65 and ≤75 years old
Hematologic Malignancies
Acute Leukemias, Acute myeloid leukemia (AML), Acute lymphoblastic leukemia (ALL)/lymphoma, Biphenotypic/Undifferentiated/Prolymphocytic Leukemias, Myelodysplastic syndrome, Chronic myelogenous leukemia, Minimal Residual Disease (MRD) positive leukemia, Leukemia or Myelodysplastic Syndromes (MDS) in aplasia, Myeloproliferative neoplasms/myelofibrosis, Relapsed large-cell lymphoma, mantle-cell lymphoma and Hodgkin lymphoma, Burkitt's lymphoma, Relapsed T-cell lymphoma, Natural Killer cell malignancies, Relapsed chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma, follicular lymphoma, Lymphoplasmacytic lymphoma, Relapsed multiple myeloma, Bone marrow failure syndromes, Clinics and Surgery Center (CSC)
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MT2011-28R: Assessment of Allogeneic Hematopoietic Stem Cell Transplantation in Medicare Beneficiaries with Myelodysplastic Syndrome and Related disorders - Part I.

Daniel Weisdorf, M.D.
NA
This study is NOT accepting healthy volunteers
NCT01166009
Allogeneic Stem Cell Transplantation, Autologous Stem Cell Transplantation, Blood Cancers, Solid Tumors
CIBMTR, Clinics and Surgery Center (CSC), Data Collection, NMDP, Stem Cell Transplantation
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APOL1 Long-term Kidney Transplantation Outcomes Network

The National Institutes of Health (NIH)-sponsored collaborative APOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO) is charged with prospectively assessing the effects of renal-risk variants (RRVs) in the apolipoprotein L1 gene (APOL1) on outcomes for kidneys from donors with recent African ancestry and the recipients of their kidneys, after deceased- and living-donor renal transplantation. For the purposes of APOLLO, recent African ancestry is defined as individuals with similar genetic make-up to those currently residing in Africa. APOLLO will also study the impact of APOL1 RRVs on the health of living kidney donors with recent African ancestry.

Samy Riad
NA
This study is NOT accepting healthy volunteers
NCT03615235
STUDY00007354
Kidney Disease, Chronic, Kidney Diseases, Kidney Failure
Apolopoprotein L1 gene (APOL1), Association of Organ Procurement Organizations (AOPO), Kidney Donor, Kidney Transplantation, Kidney Transplantation Outcomes Network, United Network for Organ Sharing (UNOS)
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A Partially-Blind, Randomized, Parallel-Group Dose Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFactTM (Aerosolized SF-RI 1) Administered by nCPAP versus nCPAP alone in the Treatment of Preterm Infants at Risk for Worsening Respiratory Distress Syndrome

A Partially-Blind, Randomized, Controlled, Parallel-Group Dose Ranging Study to Determine the Efficacy, Safety and Tolerability of AeroFactTM (SF-RI 1 surfactant for inhalation combined with a dedicated drug delivery system) in Preterm Infants at Risk of Worsening Respiratory Distress Syndrome. To determine an optimal dose of AeroFact™ administered to preterm infants on nCPAP or nIMV vs. nCPAP or nIMV alone in reducing the incidence of intubation/cannulation and bolus surfactant instillation in the first 7 days after birth. To evaluate pulmonary outcomes and respiratory resource utilization at 3, 6, 9, and 12 months PMA

Catherine Bendel
All
26 Weeks to 31 Weeks old
Phase 2
This study is NOT accepting healthy volunteers
NCT03969992
STUDY00007630
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Inclusion Criteria:

• Parental consent obtained prior to study procedures being performed (pre-natal consent is allowed)
• 26 0/7 to 30 6/7 weeks of gestational age
• Weight <2.0 Kg
• Respiratory Severity Score (RSS) 1.4-2.0
Exclusion Criteria:

• Apgar score less than or equal to 5 at five minutes after birth
• Need for chest compressions or administration of epinephrine or bicarbonate in the delivery room
• Premature rupture of membranes (PROM) > 14 days
• Need for intubation and/or mechanical ventilation prior to enrollment
• Active pneumothorax requiring chest tube
• Significant congenital anomaly, chromosomal abnormality
• Concomitant treatments with inhaled nitric oxide
Drug: AeroFact, Other: nCPAP
Respiratory Distress Syndrome in Premature Infant
Respiratory Distress Syndrome, BPD, surfactant, Preterm Infant
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COG AOST2121 - An Open-Label Phase 2 Study of Maintenance Therapy with OST31-164 After Resection of Recurrent Osteosarcoma (OST-164-01)

This phase II trial studies OST31-164 as a single agent every 3 weeks for 48 weeks, with 4 doses constituting 1 treatment cycle (12 weeks per cycle). Each patient will receive treatment at a dose of 1x109 CFU until Week 48 or until disease progression, unacceptable toxicity, or the patient meets any other treatment discontinuation criteria. Following treatment discontinuation, all patients will enter a 3-year survival follow-up period. The primary endpoints are disease control during the first 12 months after enrollment and safety assessments (adverse events [AEs], physical examinations, clinical laboratory tests, vital sign measurements, performance status, and tests to monitor for the persistence of Lm).

Emily Greengard
All
12 Years to 39 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04974008
STUDY00013667
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Inclusion Criteria:

• Note: Patients enrolled on AOST2031 are eligible for enrollment in the present study. Patients are eligible to be included in the study only if all the following criteria apply: Age and Weight
• Between 12 years of age and 39 years of age at the time the Informed Consent/ Assent form is signed.
• Weight at least 40 kg. Diagnosis
• Has histologic confirmation of osteosarcoma at diagnosis.
• Has at least one episode of disease recurrence in the lungs without limitation on the number of episodes of recurrence as long as the following criteria are met:
• Surgical resection of all possible sites of suspected pulmonary metastases to achieve a complete remission within 8 weeks prior to study enrollment
• Pathological confirmation of osteosarcoma from at least one resected tumor.
• Patients with no clinical evidence of osteosarcoma at the time of resection will not require radiographic confirmation of complete remission for enrollment Note: The definition of complete resection is: gross resection of all disease as per the operating surgeon. (For further details see https://members.childrensoncologygroup.org/files/Disc/surgery/handbooks/OsteoBoneHandb ook.pdf) Performance Status
• Patient must have a performance status corresponding to ECOG scores of 0, 1, or 2. Use Karnofsky scale for patients > 16 years of age and Lansky scale for patients < years of age Prior Therapy
• Patient must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, radiotherapy, or surgery prior to entering this study. Organ Function Requirements
• Patient has adequate organ function as defined below: a. Hematological: i. Absolute neutrophil count (ANC) is at least 1,000/µL without transfusion or growth factor support. ii. Platelet count ≥ 50,000/µL without transfusion or growth factor support. b. Adequate renal function defined as: i. Creatine clearance or radioisotope glomerular filtration rate (GFR) > 70 mL/min/1.73 m2 or ii. A serum creatine based on age/gender as follows: Maximum Serum Creatinine (mg/dL) Age: 12 to < 13 years Male :1.2 Female:1.2 Age:13 to < 16 years Male :1.5 Female:1.4 Age: ≥ 16 years Male :1.7 Female:1.4 Note: the threshold for creatinine values in this table were derived from the Schwartz formula for estimating GFR. c. Adequate liver function defined as: i. Total bilirubin < 1.5 x upper limit of normal (ULN) for age ii. Serum glutamic-pyruvic transaminase (SGPT) / alanine aminotransferase (ALT) < 110 U/L (for the purpose of this study the ULN for SGPT is 45 U/L) iii. Serum albumin > 2 g/dL d. Adequate coagulation i. International normalized ratio (INR) or prothrombin time (PT) < 1.5 x ULN unless patient is receiving anticoagulant therapy as long as PT or INR is within therapeutic range of intended use of anticoagulants. ii. Activated partial thromboplastin time (aPTT) < 1.5 x ULN unless patient is receiving anticoagulant therapy as long as aPTT is within therapeutic range of intended use of anticoagulants. e. Adequate cardiac function defined as: i. Shortening fraction of > 27% by echocardiogram, or ii. Ejection fraction of > 50% by radionuclide angiogram or echocardiogram f. Adequate pulmonary function defined as: i. No evidence of dyspnea at rest, no history of exercise intolerance, and a pulse oximetry of > 94%. g. Central nervous system (CNS) function defined as: i. Patients with a known seizure disorder may be enrolled if on anticonvulsants and/or are well-controlled. ii. CNS toxicity including peripheral neuropathy < Grade 2.
• Patient and/or patient's parent or legal guardian must be capable of understanding the investigational nature, potential risks, and benefits of the study. The patient and/or the parent or legal guardian must sign a written informed consent. Age-appropriate assent will be obtained per institutional guidelines. Contraception: Female patients :
• A female patient is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
• Not a woman of childbearing potential (WOCBP) as defined in the protocol OR
• A WOCBP who agrees to follow the contraceptive guidance in the protocol during the treatment period and for at least 120 days after the last dose of study treatment.
• A female patient of childbearing potential must have a negative urine or serum pregnancy test within 72 hours prior to receiving any dose of study treatment. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. Male patients:
• A male patient is eligible to participate if he agrees to follow the contraceptive guidance in the protocol during the study treatment period and for at least 120 days after the last dose of study treatment.
Exclusion Criteria:

• Has clinically evident metastatic or recurrent disease.
• Has concurrent pulmonary recurrence and local recurrence at the primary tumor site.
• Has primary refractory disease with progression of the primary tumor on initial-therapy.
• Has CNS or any extrapulmonary disease involvement at the time of the most recent episode of disease recurrence proceeding enrollment.
• Has active infection requiring systemic therapy or is dependent on or is currently receiving systemic antibiotics that cannot be discontinued before dosing. (Note: Patients who discontinue an antibiotic prior to dosing must wait at least 5 half-lives after the last dose of antibiotic before receiving any OST31-164 infusion). Inhaled prophylactic PJP (pneumocystis jiroveci pneumonia) treatment is acceptable per Investigator discretion.
• Is currently dependent on or has received corticosteroids within the past 4 weeks (topical corticosteroids and occasional inhaled corticosteroids are allowed).
• Is currently participating in or has participated in a study of an investigational agent or is using an investigational device within 4 weeks of the first dose of treatment.
• Has a history of other active malignancy for < 2 years prior to enrollment. Basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy or is felt by the Investigator to be at low risk for recurrence is allowed.
• Has an active autoimmune disease requiring systemic treatment within the past 3 months or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency) is not considered a form of systemic treatment. Patients with vitiligo or resolved childhood asthma/atopy would be an exception to this rule. Patients who require intermittent use of bronchodilators or local steroid injections will not be excluded from the study. Patients with hypothyroidism stable on hormone replacement or Sjogren's syndrome will not be excluded from the study.
• 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 study treatment.
• Has a known allergy to any component of the study treatment(s) formulations.
• Has a contraindication (e.g., sensitivity/allergy) to both trimethoprim/ sulfamethoxazole and amoxicillin.
• Has contraindication to administration of NSAIDs.
• Is currently receiving or will be receiving any chemotherapy, including PI3K inhibitors, during the treatment phase.
• Has had a prior monoclonal antibody therapy within 2 weeks prior to study Day 1.
• Requires or anticipates requiring tumor necrosis factor (TNF) blocking agent (e.g., infliximab) therapy for diagnosis of rheumatologic disease or inflammatory bowel disease (e.g., ankylosing spondylitis, Crohn's disease, plaque psoriasis, psoriatic arthritis, rheumatoid arthritis, or ulcerative colitis).
• Has previous history of listeriosis.
• Has a known history of human immunodeficiency virus (HIV) (HIV 1/2 antibodies).
• Has known active hepatitis B (e.g., HBsAg reactive) or hepatitis C (e.g., HCV RNA [qualitative] is detected).
• Has received a live vaccine within 30 days prior to Study Day 1.
• Patient is or has an immediate family member (spouse, children, or parent) who is directly involved with this study or is employed by the investigational site or Sponsor, unless prospective Institutional Review Board (IRB) approval (by chair or designee) is given allowing exception to this criterion for a specific patient. Regulatory Requirements:
• All patients and/or their parents or legal guardians must sign a written informed consent.
• All institutional, FDA, and NCI requirements for human studies must be met.
Drug: OST31-164
Bone Cancer
Osteosarcoma, Bone Cancer
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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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A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment Participants will be randomized to placebo or Crinecerfont for 28 weeks followed by an open-label period where all participants will receive Crinecerfont for 24 weeks. The purpose of this research study in children (ages 2 to 17 years) with CAH are: • To study whether crinecerfont can lower high levels of adrenal androgens (male hormones) and high doses of glucocorticoid medication • To study whether improving high androgen levels and high glucocorticoid doses can lead to improvements in CAH • To learn about what happens to crinecerfont in the body by measuring levels in your child’s body after starting the study drug. • To study the safety and tolerability of crinecerfont The study will last for about 60 weeks with 14 study visits. The main activities in this study are: physical exams, height and weight measurements, vital signs, study drug dosing, glucocorticoid dose adjustments, blood draws, saliva and urine tests, electrocardiogram (ECG) testing, x-ray for bone age, ultrasound of testes (boys), and questionnaires.

Kyriakie Sarafoglou
Phase III
This study is NOT accepting healthy volunteers
NCT04806451
STUDY00012858
Congenital Adrenal Hyperplasia
21-hydroxylase deficiency, classic congenital adrenal hyperplasia (CAH)
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