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290 Study Matches

MT2018-46: Longterm Follow-up of Subjects with Cerebral Adrenoleukodystrophy who were Treated with Lenti-D Drug Product

This is a multi-center, long-term safety and efficacy follow-up study for subjects with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in parent clinical studies. Lenti-D Drug Product is defined as an autologous CD34+ cell-enriched population that contains cells transduced with Lenti-D lentiviral vector encoding the human adrenoleukodystrophy protein. In parent studies, male subjects with CALD are infused on a single occasion with Lenti-D Drug Product, and then followed for 24 (±1) month for safety and efficacy. The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) recommend long-term follow-up for subjects treated with gene therapy drug products to monitor for selected adverse events (AEs), as well as durability of clinical response. Therefore, after subjects have completed the parent clinical studies, they will be asked to participate in a long-term follow-up Study LTF-304, in which they will be followed every 6 months through 5 years post-drug product infusion, and then annually through 15 years post-drug product infusion. Safety evaluations will include documentation of drug product-related AEs, all serious adverse events (SAEs) regardless of attribution to the drug product, CALD-related ≥Grade 2 AEs, and integration site analysis for the detection of clonal dominance through 15 years post drug product infusion, as well as archiving for RCL testing through 5 years post‑drug product infusion. Efficacy evaluations will include CALD disease-specific assessments, primarily major functional disabilities and brain MRI, with additional exploratory assessments for change in Loes score, Loes pattern, neurologic function score (NFS), very long chain fatty acids (VLCFA), intelligence quotient (IQ), and health related quality of life (HRQoL) assessment. To monitor pharmacodynamics, vector copy number in peripheral blood (PB VCN; vector copies per diploid genome [c/dg]) and transgenic protein expression of adrenoleukodystrophy protein (ALDP) in peripheral blood will be measured at designated study visits. There is no designated Data Monitoring Committee (DMC) for Study LTF-304; however, the review of safety data for this study, including AEs, SAEs and relevant laboratory values, may be performed by the DMC convened for the parent study in which the subject(s) originally participated.

Paul Orchard
Male
Not specified
NA
This study is NOT accepting healthy volunteers
NCT02698579
STUDY00005330
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Inclusion Criteria:

• Provision of written informed consent for this study by the participant or participant's parent(s)/ legal guardian(s) and written informed assent by participant, if applicable
• Have received Lenti-D Drug Product in a parent clinical study
• Able to comply with study requirements
Exclusion Criteria:

• There are no exclusion criteria for this Study
Genetic: Lenti-D
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
Adrenoleukodystrophy, X-linked Adrenoleukodystrophy, Hematopoietic Stem Cells
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MT2019-01: Adrenoleukodystrophy National Registry Study (ALD) and Biobank

In this protocol, we will enroll pediatric, adolescent and adult patients diagnosed with adrenoleukodystrophy (ALD). These patients will include probands diagnosed by newborn screening and their relatives subsequently diagnosed, as well other patients who are diagnosed with ALD due to other presenting signs and symptoms and subsequently were confirmed to have ALD. We will ask consenting subjects to provide a medical history (with verification via medical records), to participate in a semi-annual health survey and provide consent to collect biospecimens. The overarching goal of this work is to engage with families affected by ALD and to assemble a resource of clinical, medical, and biological data that will allow of to better understand the natural history of ALD, and how this is affected by newborn screening. The initial focus will be on patients within Minnesota, but participation will be open to any family interested in the study, as this will be web-based. This registry and biobank, together with other research conducted in tandem, will possibly provide information describing the natural history of ALD and outcomes with interventions. It is anticipated that the data collected will further our understanding of the natural history of the disease, basic biology of adrenoleukodystrophy, diagnosis and outcomes. Ultimately, this research may lead to new avenues for early diagnosis and development of safer and more effective therapies for ALD.

Ashish Gupta
All
Not specified
NA
This study is NOT accepting healthy volunteers
NCT03789721
STUDY00003605
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Inclusion Criteria
• Age 0 - 100
• ALD patients or family member meeting any of the following criteria:
• Any patient diagnosed with ALD (confirmed by positive VLCFA testing and/or genetic mutation).
• Known or presumed mutation with ALD based on pedigree or confirmed mutation in ABCD1 gene
• Participants living in the United States and territories Exclusion Criteria
• Patients diagnosed with ALD who lack the capacity to consent/assent AND do not have a designated legally authorized representative or guardian.
• Patients who have undergone BMT or other cellular therapy .
• Patients not fluent in English who are unable to consent in-person at the BMT Journey Clinic.
• Patients who are illiterate
• Patient determined by the PI or designee to be unlikely to complete required study components (due to language barriers, compliance issues, etc.)
Other: Medical Record Abstraction, Other: Biospecimen Sample Collection
ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
Registry, VLCFA, ABCD1, X-chromosome
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A Phase 2/3, prospective, open-label trial evaluating the efficacy, safety, and pharmacokinetics of remimazolam for intravenous sedation in paediatric patients undergoing diagnostic and/or therapeutic procedures

To assess the efficacy of intravenous (IV) remimazolam in inducing and maintaining suitable sedation levels for paediatric patients undergoing diagnostic and/or therapeutic procedures

Kumar Belani
All
up to 17 Years old
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT04851717
STUDY00012859
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Inclusion Criteria:

• Signed informed consent form and/or assent and willingness of patient and parent(s) to participate in the trial.
• In US sites: Paediatric male or female patients, aged ≥3 and <18 years scheduled to undergo a diagnostic or therapeutic procedure, which is medically indicated and independent from the trial.
• In European sites: Paediatric male or female patients, aged full term birth to <18 years scheduled to undergo a diagnostic or therapeutic procedure, which is medically indicated and independent from the trial.
• Maximum planned duration of procedure: 2 hours
• ASA Physical Status I-III
• Planned spontaneous breathing during sedation
• A female who is of child bearing potential (i.e. after menarche) and sexually active must use a highly effective method of birth control during the trial period (from the time of consent until all specified observations are completed)
• Negative pregnancy test at screening and on treatment day -
Exclusion Criteria:

• Emergency procedures
• Condition/procedure that requires planned airway control via endotracheal tube or LMA/IGEL insertion
• Cranio-facial malformation, which would severely limit the possibilities for emergency airway rescue
• Other abnormalities relating to the airway (including large tonsils and anatomical abnormalities of upper airway or lower airway) which may compromise emergency airway rescue
• Known hypersensitivity to benzodiazepines, flumazenil, dextran or any of the ingredients of the drug product
• Known paradoxical reactions to benzodiazepines
• History of sleep apnoea
• Active respiratory failure
• Active neuromuscular disease
• Active cardiac failure
• Active hepatic failure
• Breast feeding females
• Prohibited medication
• Any patient judged by the Principal Investigator (PI) or Sub-Investigator to be inappropriate for the trial for any other reason
Drug: Remimazolam
Pediatric ALL
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MT2019-41: A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects with Fanconi Anemia Subtype A

The objective of this study is to assess the therapeutic efficacy of a hematopoietic gene therapy consisting of autologous CD34+ enriched cells transduced with a LV carrying the FANCA gene in subjects with FA-A.

Margaret MacMillan, MD
All
1 Year and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04248439
STUDY00008719
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Inclusion Criteria:

• Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent
• Patients of the complementation group FA-A
• Minimum age: 1 year and a minimum weight of 8 kg
• At least 30 CD34+ cells/μL are determined in one bone marrow (BM) aspiration within 3 months prior to CD34+ cell collection OR (see subsequent criterion)
• If the number of CD34+ cells/ μL in BM is in the range of 10-29, peripheral blood (PB) parameters should meet two of the three following criteria:
• Hemoglobin: ≥11g/dL
• Neutrophils: ≥900 cells/μL
• Platelets: ≥60,000 cells/μL
• Provide informed consent in accordance with current legislation
• Women of childbearing age must have a negative urine pregnancy test at the baseline visit, and accept the use of an effective contraception method during participation in the trial
Exclusion Criteria:

• Subjects with an available and medically eligible HLA-identical sibling donor.
• Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities other than those reported as variant(s) of normal in BM aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the subject commences the stem cell mobilization/collection procedures of the clinical trial.
• Subjects with somatic mosaicism associated with stable or improved counts in all PB cell lineages. (If T-lymphocyte chromosomal fragility analysis indicates potential mosaicism, a medically significant decrease (≥1 NCI CTCAE grade) in at least one blood lineage over time must be documented to enable eligibility, as should <5% resistance of bone marrow colony forming cells (CFCs) to 10nM MMC; whenever possible potential mosaicism should also be evaluated by gene sequencing of MMC-resistant CFCs).
• Lansky performance status ≤60%.
• Any concomitant disease or condition that, in the opinion of the Principal Investigator, renders the subject unfit to participate in the study.
• Pre-existing sensory or motor impairment ≥grade 2 according to the criteria of the NCI.
• Pregnant or breastfeeding women.
• Hepatic dysfunction as defined by either:
• Bilirubin >3.0 × the upper limit of normal (ULN) or
• Alanine aminotransferase (ALT) > 5.0 × ULN or
• Aspartate aminotransferase (AST) > 5.0 × ULN For subjects with bilirubin, ALT or AST above ULN, a workup to identify the etiology of liver abnormality should be conducted prior to confirmation of eligibility as stipulated in exclusion criterion 5, including evaluation of viral hepatitis, iron overload, drug injury or other causes.
• Renal dysfunction requiring either hemodialysis or peritoneal dialysis.
• Pulmonary dysfunction as defined by either:
• Need for supplemental oxygen during the prior 2 weeks in absence of acute infection or
• Oxygen saturation by pulse oximetry <90%.
• Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years.
• Subject is receiving androgens (i.e. danazol, oxymetholone).
• Subject is receiving other investigational therapy for treatment/prevention of FA-associated bone marrow failure.
Biological: RP-L102
Fanconi Anemia Complementation Group A
anemia, bone marrow failure, gene therapy
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Theta Burst Stimulation for Headaches after Traumatic Brain Injury

The primary objective of this study is to investigate the safety and efficacy of theta burst stimulation (TBS) for the management of post-traumatic headaches to improve outcomes and quality of life for individuals who have suffered a traumatic brain injury (TBI). To improve tolerability and logistical burden, we have developed a novel design whereby participants will receive three doses of TBS on alternate days of the week. This design will allow us to assess efficacy while leveraging an accelerated treatment course (nine stimulation sessions per week). We have three specific aims: Specific Aim 1. To determine the efficacy and safety of TBS for the treatment of post-traumatic headache among individuals who have sustained a mild TBI. Hypothesis 1a: TBS will be safe, well-tolerated, and reduce the number of headache days. Hypothesis 1b: TBS will improve function and quality of life outcomes. Specific Aim 2: To determine the efficacy and safety of an accelerated time-course of TBS for the management of post-traumatic headache. Hypothesis 2a: The accelerated-time course will be safe, welltolerated, and improve quality of life outcomes. Hypothesis 2b: The accelerated time-course will produce greater and faster improvement in headache symptoms than that reported in the literature for standard repetitive transcranial magnetic stimulation (rTMS) protocols. Specific Aim 3: To examine the durability of treatment response to accelerated TBS during a one-month observational period. Hypothesis 3: Accelerated TBS will result in enduring treatment response of posttraumatic headache symptoms over the follow-up period.

Cristina Albott
18 Years and over
NA
This study is NOT accepting healthy volunteers
NCT04906603
STUDY00013016
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Inclusion Criteria:
? Veterans receiving services through the MVAHCS; ? History of mild to moderate TBI according to VA/DoD Clinical Practice Guidelines (2009); ? Post-traumatic headaches defined by International Classification for Headache Diagnosis 3rd Edition (ICHD-3) guidelines with the following criteria present: o Headaches developing within seven days following trauma or injury to the head and/or neck o Headaches persisting beyond three months; ? Chronic daily headaches defined by clinical standards with the following criterion present: o 15 or more headache days per month; ? Men and women 18-75 years of age; ? Possess a smartphone and agree to use the EMA application on their personal device; ? Capable and willing to provide voluntary informed consent
Exclusion Criteria:
? History of severe TBI according to VA/DoD Clinical Practice Guidelines (2009); ? Current (within six months of enrollment) psychosis and mania; ? Current (within one month of enrollment) substance dependence: o Does not include dependence on opioids for chronic pain when the medication is taken as prescribed; ? Personal history of epilepsy or seizure disorder: o Does not include seizures therapeutically-induced by electroconvulsive therapy (ECT) or identified as a single seizure event (based on the principal investigator?s judgement); ? Metal particles in the eye or head (exclusive of the mouth) (e.g., shrapnel, fragments from welding or metalwork, etc.); ? Implanted medical device controlled by physiologic signals (e.g., pacemakers, defibrillators, etc.) or implanted medical device above the clavicle (e.g., aneurysm clips, shunts, stimulators, cochlear implants, electrodes, etc.); ? Significant neurological disorder/injury or abnormal structural brain imaging that would impact risk (based on the principal investigator?s judgement and research literature); ? Unstable physical disease (e.g., severe heart disease); ? Current use of medications with significant potential for lowering seizure threshold; ? Current benzodiazepine usage at a dose higher than 3mg of lorazepam or equivalent; ? ECT or cortical energy exposure within one month of enrollment (including participation in any other neuromodulation treatments or studies); ? Current (within one month of enrollment) participation in another interventional study that would impact the results of this research; ? Inadequate communication (e.g., language barrier); ? Women who are pregnant, trying to become pregnant, or breastfeeding; ? Women of childbearing age/potential who are not using a medically-accepted form of contraception when sexually active
Brain & Nervous System, Brain Injuries, Traumatic, Post-Traumatic Headache, Quality of Life, Transcranial Magnetic Stimulation
TBI, TMS, headache
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MT2019-06: A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.

Evaluate the efficacy of treatment with bb1111 (also known as LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD).

Ashish Gupta
All
2 Years to 50 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04293185
STUDY00006923
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Inclusion Criteria:

• Have a diagnosis of SCD, with either βS/βS, βS/β0, or βS/β+ genotype.
• Be ≥2 and ≤50 years of age at time of consent.
• Weigh a minimum of 6 kg.
• Have a Karnofsky performance status of ≥60 (≥16 years of age) or a Lansky performance status of ≥60 (<16 years of age).
• Be treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on sickle cell disease history.
• In the setting of appropriate supportive care measures (e.g., pain management plan), have experienced at least 4 protocol-defined VOEs in the 24 months prior to informed consent.
• Have either experienced HU failure at any point in the past or must have intolerance to HU (intolerance is defined as the patient being unable to continue to take HU per PI judgment).
• Female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from Screening to at least 6 months after drug product infusion.
• Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s).
Exclusion Criteria:

• Subjects for whom allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate per PI judgment and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available.
• Severe cerebral vasculopathy, defined by any history of overt ischemic or hemorrhagic stroke, a history of abnormal transcranial Doppler (TCD) or TCD imaging (TCDI) for subjects ≤ 16 years of age (e.g. TCD velocity >200 cm/sec) requiring ongoing chronic transfusions, a Screening TCD or TCDI velocity > 200 cm/sec (central read), a Screening MRA showing > 50% stenosis or occlusion in the circle of Willis (central read), or a Screening MRA showing the presence of Moyamoya (central read).
• Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B, hepatitis C, human T-lymphotropic virus-1 (HTLV-1), active syphilis.
• Clinically significant, active bacterial, viral, fungal, or parasitic infection
• Advanced liver disease, such as
• clear evidence of liver cirrhosis, active hepatitis or significant fibrosis (based on MRI or liver biopsy)
• liver iron concentration ≥15 mg/g unless liver biopsy shows no evidence of cirrhosis, active hepatitis or significant fibrosis
• Inadequate bone marrow function, as defined by an absolute neutrophil count of <1×10^9/L (<0.5×10^9/L for subjects on hydroxyurea treatment) or a platelet count <100×10^9/L.
• Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients.
• Patients needing therapeutic anticoagulation treatment during the period of conditioning through platelet engraftment
• Unable to receive pRBC transfusion.
• Prior receipt of an allogeneic transplant.
• Prior receipt of gene therapy.
• Any prior or current malignancy or immunodeficiency disorder, except previously treated, non-life threatening, cured tumors such as squamous cell carcinoma of the skin.
• Immediate family member with a known or suspected Familial Cancer Syndrome.
• Female subject is breastfeeding, pregnant or will attempt to become pregnant from Screening to at least 6 months after drug product infusion.
• Any other condition that would render the subject ineligible for HSCT.
• Participation in another clinical study with an investigational drug within 30 days of screening.
• Presence of a chromosomal abnormality or genetic mutation that may put the subject at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) per Investigator's judgment.
• Presence of genetic mutations that result in the inactivation of 2 or more α-globin genes
Genetic: bb1111
Sickle Cell Disease
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MT2014-10C : Allogeneic Hematopoietic Stem Cell Transplant for Patients with High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders

Ashish Gupta
All
up to 55 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02179359
1407M52125
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Inclusion Criteria:

• Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
• Acceptable stem cell source identified
• Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
• Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
• Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal
• Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%
Exclusion Criteria:

• active, uncontrolled infection
• pregnant or breastfeeding
• HIV positive
Drug: Reduced Toxicity Ablative Regimen, Drug: Reduced Intensity Preparative Regimen, Drug: Myeloablative Preparative Regimen
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Stem Cell Transplant, Clinics and Surgery Center (CSC)
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A Randomized, Open Label, Phase III Trial to Evaluate the Efficacy and Safety of Palbociclib + Anti-HER2 Therapy + Endocrine Therapy vs. Anti-HER2 Therapy + Endocrine Therapy After Induction Treatment for Hormone Receptor Positive (HR+)/HER2-Positive Metastatic Breast Cancer

Phase III
This study is NOT accepting healthy volunteers
NCT02947685
201707881
Estrogen Receptor Positive Breast Cancer, HER-2 Positive Breast Cancer
HER2+, HR+, breast cancer, malignant tumor of the breast, metastatic breast cancer
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MT2005-25 Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

To determine the incidence of engraftment (defined as achieving donor derived neutrophil count >500/uL by day 42) in young children with leukemia or myelodysplastic syndrome undergoing a partially matched single unit umbilical cord blood transplant (UCBT) after a myeloablative preparative regimen consisting of busulfan, melphalan and fludarabine.

Christen Ebens
All
up to 3 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT00357565
0511M77206
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Inclusion Criteria:

• Matched sibling donor (HLA 8/8), if available, or a unrelated partially HLA matched single unit based on the following priority:
• 1st priority: 4/6 matched unit, cell dose >5 x 10-7 nucleated cells/kg
• 2nd priority: 5/6 matched unit, cell dose > 4 x 10-7 nucleated cells/kg
• 3rd priority: 6/6 matched unit, cell dose > 3 x 10-7 nucleated cells/kg
• Patients aged ≤ 3 years at diagnosis (not age of transplant) with hematological malignancy as detailed below:
• Acute myeloid leukemia: high risk CR1 as evidenced by:
• High risk cytogenetics t(4;11) or other MLL rearrangements; chromosome 5, 7, or 19 abnormalities; complex karyotype (>5 distinct changes); ≥ 2 cycles to obtain complete response (CR); CR2 or higher; Preceding myelodysplastic syndrome (MDS); All patients must be in CR or early relapse (i.e., <15% blasts in BM).
• Acute lymphocytic leukemia: high risk CR1 as evidenced by: High-risk cytogenetic: t(4;11) or other MLL rearrangements; hypodiploid; t(9;22); >1 cycle to obtain CR; CR2 or higher; All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
• Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics. Blasts must be < 10% by a representative bone marrow aspirate morphology.
• Persistent or rising minimal residual disease (MRD) after standard chemotherapy regimens: Patients with evidence of minimal residual disease at the completion of therapy or evidence of rising MRD while on therapy. MRD will be defined by either flow cytometry (>0.1% residual cells in the blast gate with immune phenotype of original leukemic clone), by molecular techniques (PCR or FISH) or conventional cytogenetics (g-banding).
• New Leukemia Subtypes: A major effort in the field of pediatric 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 high risk features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
• Recipients must have a Lansky score ≥ 50% and have acceptable organ function defined as:
• Renal: glomerial filtration rate > 60ml/min/1.73m^2
• Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal,
• Pulmonary function: oxygen saturation >92%
• Cardiac: left ventricular ejection fraction > 45%.
• Voluntary written informed consent before performance of any study-related procedure not part of normal medical care.
Exclusion Criteria:

• Active infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days).
• History of HIV infection or known positive serology
• Myeloablative transplant within the last 6 months.
• Evidence of active extramedullary disease (including central nervous system leukemia).
Biological: filgrastim, Drug: busulfan, Drug: cyclosporine, Drug: fludarabine phosphate, Drug: melphalan, Drug: mycophenolate mofetil, Procedure: umbilical cord blood transplantation
Leukemia, Myelodysplastic Syndromes, Childhood Acute Myeloid Leukemia in Remission, Recurrent Childhood Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Childhood Acute Lymphoblastic Leukemia in Remission, Previously Treated Myelodysplastic Syndrome, Secondary Myelodysplastic Syndrome, Refractory Anemia With Excess Blasts in Transformation, Refractory Anemia With Excess Blasts, Refractory Anemia, De Novo Myelodysplastic Syndrome, Childhood Myelodysplastic Syndrome
MDS, AML
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An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants with Type 2 Gaucher Disease

This is a study to assess the safety and efficacy of PR001A, an Aden-associated (AAV9) viral vector to treat neuronopathic Gaucher disease type 2 (GD2) in infants. PRA001A will be administered via suboccipital injection to the cisterna magna during a single neurosurgical session. GD2 is a fatal disease of early infancy that does not have any therapeutic options beyond palliative care. This study will enroll infants 0-24 months of age.

Chester Whitley, MD, PhD
All
up to 24 Months old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04411654
STUDY00008823
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Inclusion Criteria:

• Bi-allelic GBA1 mutations consistent with a diagnosis of GD2 confirmed by the central laboratory.
• Clinical diagnosis of GD2
• Parent/legal guardian has the ability to understand the purpose and risks of the study and provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations.
• Patient has a reliable informant (i.e., parent/legal guardian) willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities (including providing input into the rating scales).
Exclusion Criteria:

• Diagnosis of a significant CNS disease other than GD2 that may be a cause for the patient's GD symptoms or may confound study objectives.
• Achieved independent gait.
• Severe peripheral symptoms of GD which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
• Concomitant disease, condition, or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
• Use of any GD treatment-related substrate reduction therapy.
• Use of strong inhibitors or inducers of cytochrome P450 3A4 (CYP3A4) or P-glycoprotein (P-gp) medications, herbals, or over-the-counter agents.
• Any type of prior gene or cell therapy.
• Live vaccine Immunizations within 4 weeks, or non-live vaccines within 2 weeks prior to the start of required immunosuppressive regimen.
• Use of blood thinners. Antiplatelet therapies are acceptable if the patient is medically able to temporarily stop them from 7 days prior to dosing and through at least 48 hours after the intracisternal injection and lumbar puncture.
• Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified (topical or inhaled preparations for dermatological conditions or asthma are allowed).
• Participation in another investigational drug or device study within the past 3 months.
• Brain MRI (magnetic resonance imaging) and MRA (magnetic resonance angiography) showing clinically significant abnormality deemed a contraindication to intracisternal injection.
• Clinically significant laboratory test result abnormalities assessed at screening.
• Contraindications or intolerance to radiographic visualization methods (e.g. MRI, MRA, CT), and intolerance to contrast agents used for MRI or CT scans.
• Contraindications to general anesthesia or sedation. Other protocol-defined inclusion/exclusion criteria may apply.
Biological: LY3884961, Drug: Methylprednisolone, Drug: Sirolimus, Drug: Prednisone
Gaucher Disease, Type 2
Gaucher Disease, GD, Gaucher, Type 2 Gaucher, Neuronopathic Gaucher, nGD, AAV9, GBA, Gene Therapy, Glucocerebrosidase, GBA1 mutation, Infants
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MT2013-34C: Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia: Standard of Care Considerations

Christen Ebens
All
up to 70 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02162420
1404M50183
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Inclusion Criteria:

• Aged 0 - 70 years
• Acceptable hematopoeitic stem cell donor
• Dyskeratosis Congenita (DC) with evidence of BM failure defined as:
• requirement for red blood cell and/or platelet transfusions or
• requirement for G-CSF or GM-CSF or erythropoietin or
• refractory cytopenias having one of the following three
• platelets <50,000/uL or transfusion dependent
• absolute neutrophil count <500/uL without hematopoietic growth factor support
• hemoglobin <9g/uL or transfusion dependent
• Diagnosis of DC with a triad of mucocutaneous features:
• oral leukoplakia
• nail dystrophy
• abnormal reticular skin hyperpigmentation, or
• Diagnosis of DC with one of the following:
• short telomeres (under a research study)
• mutation in telomerase holoenzyme (DKC1, TERT, TERC, NOP10, NHP2, TCAB1)
• mutation in shelterin complex (TINF2)
• mutation in telomere-capping complex (CTC1)
• Severe Aplastic Anemia (SAA) primary transplant with evidence of BM failure:
• Refractory cytopenia defined by bone marrow cellularity <50% (with < 30% residual hematopoietic cells)
• Diagnosis of SAA with refractory cytopenias having one of the following three:
• platelets <20,000/uL or transfusion dependent
• absolute neutrophil count <500/uL without hematopoietic growth factor support
• absolute reticulocyte count <20,000/uL
• Severe Aplastic Anemia (SAA) requiring a 2nd transplant
• Graft failure as defined by blood/marrow chimerism of < 5%
• Early myelodysplastic features
• With or without clonal cytogenetic abnormalities
• Adequate organ function defined as:
• cardiac: left ventricular ejection fraction ≥ 35% with no evidence of decompensated heart failure
• pulmonary: DLCO ≥30% predicted, no supplemental oxygen requirement
• renal: Glomerular filtration rate (GFR) ≥30% predicted
• Voluntary written consent
Exclusion Criteria:

• Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
• Pregnant or lactating
• Uncontrolled infection
• Prior radiation therapy (applies to SAA patients only)
• Diagnosis of Fanconi anemia based on DEB
• Diagnosis of dyskeratosis congenita with advanced MDS or acute myeloid leukemia with >30% blasts
Drug: Alemtuzumab, Drug: Fludarabine, Drug: Cyclophosphamide, Radiation: Total Body Irradiation, Biological: Stem Cell Transplant, Drug: Anti-thymocyte globulin
Dyskeratosis Congenita, Aplastic Anemia
Clinics and Surgery Center (CSC), severe aplastic anemia, Hematopoietic Stem Cell Transplant
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A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Rosnilimab (ANB030) in the Treatment of Subjects with Alopecia Areata

This research is studying a new treatment, rosnilimab, in a small number of people to learn about the safety, potential effect on alopecia areata, and how it interacts with the body. Researchers want to understand if rosnilimab may cause hair regrowth in people with alopecia areata and how it may work.

Maria Hordinsky
18 Years and over
Phase II
This study is NOT accepting healthy volunteers
NCT05205070
STUDY00014901
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Inclusion Criteria:
Men and women, adults 18- 65 years of age who are in good health Diagnosed with severe or very severe Alopecia Areata and have greater than or equal to 50% scalp hair loss Must be willing to stop using other types of medications to treat Alopecia Areata throughout the study Attend 10 visits over 28 weeks Other requirements, which the study team will review with you
Exclusion Criteria:
Subjects cannot participate in this study if they have another cause of hair loss in addition to alopecia areata. Subjects also cannot participate if they have had surgery in the last 4 weeks, if immune system is compromised, if are allergic to anything in rosnilimab, if recently used a prescription treatment for alopecia areata, if have tuberculosis, if person is pregnant or breastfeeding, or if person intends to become pregnant while part of this study. Subjects cannot participate in this study if they are currently part of another study that is using an experimental drug or device (medical tool).
Alopecia Areata, Dermatology (Skin, Hair & Nails), Immune Diseases
hair loss, Alopecia areata, PD-1 receptor, Rosnilimab
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Immunotherapy (Nivolumab or Brentuximab Vedotin) Plus Combination Chemotherapy in Treating Patients With Newly Diagnosed Stage III-IV Classic Hodgkin Lymphoma

This is a randomized Phase III study comparing the addition of nivolumab or brentuximab vedotin to a backbone of AVD chemotherapy for the initial treatment of advanced stage Hodgkin lymphoma. In addition to the primary therapeutic objective, we will conduct a series of quality of life analyses to assess the impact of each treatment regimen on patient well-being. This also studies the prognostic impact of imaging-based biomarkers like pre-treatment metabolic tumor volume and studies outcomes based on novel response assessment criteria (i.e. LYRIC and RECIL) that specifically address atypical responses observed with immunotherapy. Banking tumor tissue and blood for future translational medicine studies also.

Peter Gordon
All
12 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03907488
STUDY00015866
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Inclusion Criteria:

• All patients must have histologically confirmed newly diagnosed, previously untreated stage III or IV classical Hodgkin lymphoma (nodular sclerosing, mixed cellularity, lymphocyte-rich, or lymphocyte-depleted, or not otherwise specified [NOS]). Nodular lymphocyte predominant Hodgkin lymphoma is not eligible.
• Patients must have bidimensionally measurable disease (at least one lesion with longest diameter >= 1.5 cm) documented on the Lymphoma Baseline Tumor Assessment Form in Rave.
• Patients must have a whole body or limited whole body PET-CT scan performed within 42 days prior to registration. (A contrast-enhanced [diagnostic] CT, MRI or MR-PET is acceptable in event that PET-CT is contra-indicated, however if it is later possible to administer a PET-CT, then PET-CT is strongly preferred for the interim scan (after cycle 2) (if performed) and the EOT assessment. Otherwise, if PET-CT is not subsequently possible, then the same modality as baseline must be used throughout the trial.) NOTE: All images from PET-CT, CT, MRI or MR-PET scans performed as standard of care to assess disease (within 42 days prior to registration) must be submitted and associated radiology reports must be submitted.
• Patients must not have received any prior chemotherapy, radiation, or antibody-based treatment for classical Hodgkin lymphoma. Steroid pre-treatment is permitted.
• Patients must not have had prior solid organ transplant.
• Patients must not have had prior allogeneic stem cell transplantation.
• Patients must not have received a live vaccine within 30 days prior to planned day 1 of protocol therapy (e.g. measles, mumps, rubella, varicella, yellow fever, rabies, Bacillus Calmette-Guerin [BCG], oral polio vaccine, and oral typhoid).
• At registration, investigator must declare intent-to-treat with residual PET radiation therapy (residual PET RT- RPRT) to be administered after patient completes 6 cycles of therapy if, after end of treatment, the patient meets criteria specified for receiving RT). Patients will be stratified by investigator's intent-to-treat with residual PET RT.
• All pediatric patients (< 18 years of age) will be considered intent-to-treat with Residual PET RT at time of registration.
• Patients must have a performance status corresponding to Zubrod scores of 0, 1 or 2. Use Lansky for patients =< 17 years of age. *The conversion of the Lansky to Eastern Cooperative Oncology Group (ECOG) scales is intended for National Cancer Institute (NCI) reporting purposes only.
• Adults (age 18 or older): Creatinine clearance >= 30 mL/min, as estimated by the Cockcroft and Gault formula. The creatinine value used in the calculation must have been obtained within 28 days prior to registration. Estimated creatinine clearance is based on actual body weight. Pediatric Patients (age 12-17), the following must have been obtained within 14 days prior to registration:
• Measured or calculated creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 ml/min/1.73 m^2, or
• Serum creatinine =< 1.5 x institutional upper limit of normal (IULN), or a serum creatinine (SCr) based on age/gender as follows:
• Age < 13 maximum serum creatinine: Male 1.2 mg/dL; Female 1.2 mg/dL
• Age 13 to < 16 maximum serum creatinine: Male 1.5 mg/dL; Female 1.4 mg/dL
• Age 16-17 maximum serum creatinine: Male 1.7 mg/dL; Female 1.4 mg/dL
• Total bilirubin =< 2 x IULN (must be documented within 28 days prior to registration for adults [age 18 or older]; must be documented within 14 days prior to registration for pediatric patients [age 12-17]).
• Unless due to Gilbert's disease, lymphomatous involvement of liver or vanishing bile duct syndrome
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 3 x IULN (must be documented within 28 days prior to registration for adults [age 18 or older]; must be documented within 14 days prior to registration for pediatric patients [age 12-17]).
• Unless due to Gilbert's disease, lymphomatous involvement of liver or vanishing bile duct syndrome
• Patients must have an echocardiogram (ECHO), multigated acquisition (MUGA), or functional cardiac imaging scan with a left ventricular ejection (LVEF) fraction >= 50% or a shortening fraction of >= 27%. For all patients, the ECHO, MUGA, or functional cardiac imaging scan must be performed within 42 days prior to registration.
• Patients with known human immunodeficiency virus (HIV) infection must be receiving anti-retroviral therapy and have an undetectable or unquantifiable viral load at their most recent viral load test within 6 months prior to registration.
• Patients must not have known active hepatitis B (HBV) or hepatitis C virus (HCV) at date of registration. Patients with previously treated HBV or HCV that have an undetectable viral load within 6 months prior to registration and no residual hepatic impairment are eligible.
• Patients must not have any known central nervous system lymphoma.
• Patients must not have a history of or active interstitial pneumonitis or interstitial lung disease.
• Patients must not have had a diagnosis of inherited or acquired immunodeficiency.
• Patients must not have any known uncontrolled intercurrent illness including, but not limited to symptomatic congestive heart failure, unstable angina pectoris, hemodynamically unstable cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
• Patients must not have a condition requiring systemic treatment with either corticosteroids (> 10 mg daily prednisone equivalents) or other immunosuppressive medications within 14 days prior to registration. Inhaled or topical steroids, and adrenal replacement doses > 10 mg daily prednisone equivalents are permitted in the absence of active autoimmune disease. Steroid use for the control of Hodgkin lymphoma symptoms is allowable, but must be discontinued prior to cycle 1, day 1.
• Patients with peripheral neuropathy must have < grade 2 at date of registration.
• Patients must not have active autoimmune disease that has required systemic treatment in past 2 years (i.e., with use of disease modifying agents, immunosuppressive drugs, or corticosteroids with doses higher than prednisone 10 mg or equivalent). Autoimmune diseases include but are not limited to autoimmune hepatitis, inflammatory bowel disease (including ulcerative colitis and Crohn's disease), as well as symptomatic disease (e.g.: rheumatoid arthritis, systemic progressive sclerosis [scleroderma], systemic lupus erythematosus, autoimmune vasculitis [e.g., Wegener's granulomatosis]); central nervous system (CNS) or motor neuropathy considered of autoimmune origin (e.g., Guillain-Barre syndrome and myasthenia gravis, multiple sclerosis or glomerulonephritis). Vitiligo, alopecia, hypothyroidism on stable doses of thyroid replacement therapy, psoriasis not requiring systemic therapy within the past 2 years are permitted.
• No second prior malignancy is allowed except for adequately treated basal (or squamous cell) skin cancer, any in situ cancer or other cancer for which the patient has been disease free for two years.
• Females of childbearing potential must not be pregnant or nursing, and have a negative pregnancy test within 28 days prior to registration. Women/men of reproductive potential must have agreed to use an effective contraceptive method while receiving study drug and for women until 6 months after receiving the last dose of study drug or, for men, until 7 months after receiving the last dose of study drug. A woman is considered to be of "reproductive potential" if she has had menses at any time in the preceding 12 consecutive months. In addition to routine contraceptive methods, "effective contraception" also includes heterosexual celibacy and surgery intended to prevent pregnancy (or with a side-effect of pregnancy prevention) defined as a hysterectomy, bilateral oophorectomy or bilateral tubal ligation. However, if at any point a previously celibate patient chooses to become heterosexually active during the time period for use of contraceptive measures outlined in the protocol, he/she is responsible for beginning contraceptive measures.
• Patients must have one formalin-fixed paraffin embedded (FFPE) diagnostic tumor block or at least 1 diagnostic, 4-5 micron, hematoxylin and eosin (H&E) slide collected prior to registration and available for submission.
• Patients must be offered participation in banking for planned translational medicine and future research. With patient consent, any residuals from the mandatory tissue submission will also be banked for future research.
• Patients who can complete Patient-Reported Outcome instruments in English, Spanish, or French must complete the PROMIS Fatigue, the FACT/GOG-Ntx, and the PROMIS Global prior to registration.
• Patients who can complete Patient-Reported Outcome instruments in English, Spanish, or French must also agree to complete the PROMIS Fatigue, the FACT/GOG-Ntx, the PROMIS Global, and the PRO-CTCAE (or Ped PRO-CTCAE) at the scheduled on-study assessment timepoints.
• Patients must be informed of the investigational nature of this study and all patients and/or their parents or legal guardians (for patients < 18 years of age) must sign and give informed consent and assent (where appropriate) in accordance with institutional and federal guidelines. For participants with impaired decision-making capabilities, legally authorized representatives may sign and give informed consent on behalf of study participants in accordance with applicable federal, local, and Central Institutional Review Board Initiative (CIRB) regulations.
• Note: As a part of the Oncology Patient Enrollment Network (OPEN) registration process the treating institution's identity is provided in order to ensure that the current (within 365 days) date of institutional review board approval for this study has been entered in the system.
Drug: Brentuximab Vedotin, Drug: Dacarbazine, Drug: Doxorubicin Hydrochloride, Biological: Filgrastim, Biological: Nivolumab, Biological: Pegfilgrastim, Other: Quality-of-Life Assessment, Other: Questionnaire Administration, Radiation: Radiation Therapy, Drug: Vinblastine Sulfate
Ann Arbor Stage III Hodgkin Lymphoma, Ann Arbor Stage III Lymphocyte-Depleted Classic Hodgkin Lymphoma, Ann Arbor Stage III Mixed Cellularity Classic Hodgkin Lymphoma, Ann Arbor Stage III Nodular Sclerosis Classic Hodgkin Lymphoma, Ann Arbor Stage IIIA Hodgkin Lymphoma, Ann Arbor Stage IIIB Hodgkin Lymphoma, Ann Arbor Stage IV Hodgkin Lymphoma, Ann Arbor Stage IV Lymphocyte-Depleted Classic Hodgkin Lymphoma, Ann Arbor Stage IV Mixed Cellularity Classic Hodgkin Lymphoma, Ann Arbor Stage IV Nodular Sclerosis Classic Hodgkin Lymphoma, Ann Arbor Stage IVA Hodgkin Lymphoma, Ann Arbor Stage IVB Hodgkin Lymphoma, Classic Hodgkin Lymphoma, Lymphocyte-Rich Classic Hodgkin Lymphoma
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A Randomized, Double-blind, Placebo-controlled, Multicenter Trial, Assessing the Impact of Inclisiran on Major Adverse Cardiovascular Events in Participants With Established Cardiovascular Disease (VICTORION-2 PREVENT)

CKJX839B12302 is a pivotal, randomized, double-blind, placebo controlled, multicenter Phase III trial, designed at assessing the benefits of inclisiran sodium 300 mg s.c., administered on Day 1, Month 3 (Day 90), and every 6 months thereafter in addition to well-tolerated high-intensity statin therapy, on major adverse cardiovascular events (3P-MACE defined as first occurrence of cardiovascular death, non-fatal myocardial infarction (MI) or non-fatal ischemic stroke), in a secondary prevention cohort of atherosclerotic cardiovascular disease (ASCVD) participants with a LDL-C ≥1.8 mmol/L (70 mg/dL).

Daniel Duprez
Phase III
This study is NOT accepting healthy volunteers
NCT05030428
STUDY00015316
Atherosclerotic Cardiovascular Disease
Atherosclerotic cardiovascular disease, Clinics and Surgery Center (CSC), Dyslipidemia, Hypercholesterolemia, Hyperlipidemia, Inclisiran, KJX839, high cholesterol, siRNA
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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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MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions

Jakub Tolar, MD
All
up to 25 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02582775
1510M79481
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Inclusion Criteria:

• Diagnosis of severe form of EB characterized by collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting and by mutation analysis).
• Adequate organ function within 4 weeks of study registration defined as:
• Renal: glomerular filtration rate within normal range for age
• Hepatic: Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal
• Pulmonary: adequate pulmonary function in the opinion of the enrolling investigator
• Cardiac: left ventricular ejection fraction ≥ 45%, normal EKG or approved by Cardiology for transplant
• Sexually active participants must agree to use adequate birth control for the during the study period (from before the start of the preparative chemotherapy through 1 year post-transplant)
• Available donor per section 5: targeted MFI < 1,000 (MFI exceeding 1000 must be approved by the PI and treatment team.)
• Voluntary written consent - adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment
Exclusion Criteria:

• beta 3 laminin JEB mutants
• Active untreated systemic infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days)
• History of HIV infection
• Evidence of squamous cell carcinoma
• Pregnant or breast feeding. Females of child-bearing potential must have a negative pregnancy test prior to study registration as the agents administered in this study are Pregnancy Category C and D.
Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusions, Drug: Busulfan
Epidermolysis Bullosa
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MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG

To evaluate the ability to achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) using related and unrelated BM, PBSC, or UCB grafts following a reduced intensity conditioning regimen based on targeted-exposure busulfan, fludarabine +/- serotherapy in patients with inherited metabolic disorders and severe osteopetrosis.

Paul Orchard
All
up to 55 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02171104
1406M51542
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Inclusion Criteria:

• 0 through 55 years of age
• Adequate graft available
• Adequate organ function
• Eligible Diseases:
• Mucopolysaccharidosis Disorders:
• MPS IH (Hurler syndrome)
• MPS II (Hunter syndrome) if the patient has no or minimal evidence of symptomatic neurologic disease but is expected to have a neurologic phenotype
• MPS VI (Maroteaux-Lamy syndrome)
• MPS VII (Sly syndrome)
• Glycoprotein Metabolic Disorders:
• Alpha mannosidosis
• Fucosidosis
• Aspartylglucosaminuria
• Sphingolipidoses and Recessive Leukodystrophies:
• Globoid cell leukodystrophy
• Metachromatic leukodystrophy
• Niemann-Pick B patients (sphingomyelin deficiency)
• Niemann-Pick C subtype 2
• Peroxisomal Disorders:
• Adrenoleukodystrophy with cerebral involvement
• Zellweger syndrome
• Neonatal Adrenoleukodystrophy
• Infantile Refsum disease
• Acyl-CoA-Oxidase Deficiency
• D-Bifunctional enzyme deficiency
• Multifunctional enzyme deficiency
• Alpha-methylacyl-CoA Racmase Deficiency (AMACRD)
• Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)
• Severe Osteopetrosis (OP)
• Hereditary Leukoencephalopathy with axonal spheroids (HDLS; CSF1R mutation)
• Other Inherited Metabolic Disorders (IMD): Patients will also be considered who have other life-threatening, rare lysosomal, peroxisomal or other similar inherited disorders characterized by white matter disease or other neurologic manifestations for which there is rationale that transplantation would be of benefit, such as certain patients with Wolman's disease, GM1 gangliosidosis, I-cell disease, Tay-Sachs disease, Sandhoff disease or others.
• Voluntary written consent
Exclusion Criteria:

• Pregnancy - menstruating females must have a negative serum or urine pregnancy test within 14 days of study treatment start
• Prior myeloablative chemotherapy exposure within 4 months of the start of conditioning on this protocol (patients excluded for this reason may be eligible for other institutional protocols)
• Uncontrolled bacterial, fungal or viral infections including HIV (including active infection with Aspergillus or other mold within 30 days)
Biological: Stem Cell Transplantation, Drug: IMD Preparative Regimen, Drug: Osteopetrosis Only Preparative Regimen, Drug: Osteopetrosis Haploidentical Only Preparative Regimen, Drug: cALD SR-A (Standard-Risk, Regimen A), Drug: cALD SR-B (Standard-Risk, Regimen B), Drug: cALD HR-D (High-Risk, Regimen C), Drug: cALD HR-D (High-Risk, Regimen D)
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS, CSF1R Mutation), Inherited Metabolic Disorders
Clinics and Surgery Center (CSC), allogeneic hematopoietic cell transplantation, bone marrow transplantation, IMD, AMACRD, MNGIE, HDLS, OP, ALD
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A Phase 3, Open-label, Randomized Study of Nivolumab Combined with Ipilimumab, or with Standard of Care Chemotherapy, versus Standard of Care Chemotherapy in participants with Previously Untreated Unresectable or Metastatic Urothelial Cancer

This is a randomized, open-label, Phase 3 study comparing combination therapy of nivolumab plus ipilimumab administered every 3 weeks for up to 4 doses, followed by nivolumab monotherapy administered every 4 weeks, versus the standard of care in participants with previously untreated unresectable or metastatic urothelial cancer. In addition, a substudy of treatment with nivolumab 360mg in combination with SoC every 3 weeks for up to 6 cycles followed by nivolumab monotherapy versus SoC alone will be evaluated in cisplatin-eligible participants with previously untreated unresectable or metastatic urothelial cancer.

Gautam Jha
Phase III
This study is NOT accepting healthy volunteers
NCT03036098
STUDY00001304
Urothelial Cancer
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A Pivotal Phase 3 Trial to Evaluate the Safety and Efficacy of Clazakizumab for the Treatment of Chronic Active Antibody-Mediated Rejection in Kidney Transplant Recipients

Active antibody-mediated rejection (ABMR), especially chronic active antibody-mediated rejection (CABMR), is now recognized as the most common cause of allograft failure after a successful kidney transplant. Current standard of care anti-rejection treatments target cellular-mediated (i.e., T cell-mediated rejection (TCMR)) processes and do not affect this antibody-mediated process. Currently, there are no approved treatments for active ABMR, including CABMR. Interleukin 6 (IL-6) appears to be a critical cytokine involved in ABMR. It promotes the development and maturation of B cells to plasma cells that produce donor-specific antibodies (DSA) targeting the allograft. These DSA damage the allograft via complement and non-complement mediated pathways and induce graft endothelial cells to produce inflammatory (e.g., p-selectin, VCAM-1) and pro-thrombotic (e.g., vWF) molecules. Furthermore, IL-6 shapes the T cell immune response resulting in promotion of long-lived pro-inflammatory T helper (Th) cells (e.g., follicular Th cells, Th17, Th1, Th2) and inhibition of immune regulatory T cells (Treg), which promote allograft tolerance. This trial investigates whether clazakizumab (an anti-IL-6 monoclonal antibody (mAb)) may be beneficial for the treatment of CABMR in recipients of a kidney transplant by inhibiting the production of DSA and re-shaping T cell alloimmune responses.

Arthur Matas, MD
Phase III
This study is NOT accepting healthy volunteers
NCT03744910
STUDY00008516
Antibody-mediated Rejection
Antibody-mediated Rejection (AMR), Chronic Active, Clinics and Surgery Center (CSC)
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MT2012-11C: Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)

This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HSCT. While it will primarily be applied for the treatment of non-malignant diseases (NMD), on occasion it may be used to treat patients with malignant disorders as well.

Troy Lund
All
up to 55 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01666080
1207M17641
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Inclusion Criteria:

• Diagnosis of any disease for which a second or greater hematopoietic stem cell transplant is needed due to insufficient donor chimerism following hematopoietic recovery after previous HSCT. Determination of "insufficiency of donor chimerism" will be made by the treating transplant physician. Occasionally donor derived engraftment may be present, but sustained aplasia or failed recovery of sufficient hematopoiesis requires administration of a second graft. This intervention may be used for both situations.
• 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
• Transplantation using sufficiently matched related donors (such as matched siblings) or unrelated donors will be considered. Both granulocyte-colony stimulating factor (GCSF) stimulated peripheral blood grafts and bone marrow grafts will be considered, although bone marrow will be the priority.
• Cord blood grafts, both related and unrelated, are also eligible. As this protocol will use a reduced intensity regimen, this protocol will use the current recommendations of the University of Minnesota for choosing cord blood grafts. If a single cord blood unit cell dose is insufficient, double cord transplantation should be considered if sufficiently matched cord blood units are available. The priority of choosing cord blood donors is based on the current institutional recommendations.
• Exclusion of Metabolic Disorder or other Inherited Disorder Carrier Status from related donor and unrelated cord blood grafts as appropriate for primary disease. At the discretion of the treating transplant physician, an allograft from the previous donor may be used, if available.
• Age, Performance Status, Consent
• Age: 0 to 55 years
• Consent: voluntary written consent (adult or parental/guardian)
Exclusion Criteria:

• Previous irradiation that precludes the safe administration of an additional dose of 200 cGy of total body irradiation (TBI). Radiation Oncology will evaluate all patients who have had previous radiation therapy or TBI for approval to receive an additional 200 cGy of TBI
• Pregnant or breastfeeding
• Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
• HIV positive
• While it would be advantageous to begin therapy on this second transplant regimen > 6 months following a prior myeloablative regimen or >2 months after a reduced intensity regimen, it is recognized that there are circumstances where this may not be practical.
Drug: Busulfan, Drug: Fludarabine, Radiation: Total body irradiation, Biological: Stem cell transplant, Drug: Keppra
Hematologic Disorders, Hemoglobinopathies, Immunodeficiencies
second stem cell transplant, donor hematopoietic engraftment, hematopoietic stem cell transplantation, inherited metabolic disorder
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A Parallel-group, Two-staged, Phase 2/3, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of REC-2282 in Participants with Progressive 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.

Christopher Moertel, MD
All
12 Years and over
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT05130866
STUDY00015488
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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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A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Hormone and How Safe it is (DETECT)

The researchers are doing this study to help children and teenagers with suspected growth hormone deficiency to be able to get only one stimulation test in the future instead of two stimulation tests, as it is standard now. This one stimulation test would be the new macimorelin study test. This stimulation study test has been approved as a test on diagnosing growth hormone deficiency in adults. It is being tested in clinical trials in pediatric participants and its use in this study is investigational because it has not been approved for use in children.

All
2 Years to 17 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04786873
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Inclusion Criteria:

• Informed consent of subject, parent(s) or legally acceptable representative (LAR) of subject and child assent, if appropriate, must be obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
• Male and female pediatric subjects from 2 to less than 18 years of age at the time of signing informed consent.
• Indication for the performance of growth hormone stimulation test.
• Presence of a height measurement minimum 6 and maximum 18 months prior to screening.
Exclusion Criteria:

• Established diagnosis of a disease that is sufficient to explain growth deficiency or metabolic disorders that are also associated with short stature (e.g., Turner syndrome, skeletal dysplasia's, celiac disease, etc.).
• Ongoing growth hormone therapy.
• Presence of hypothyroidism and/or adrenal insufficiency without adequate and stable replacement therapy treatment for at least 30 days prior to first GHST.
• Treatment with drugs directly affecting the pituitary secretion of somatotropin (e.g., somatostatin analogues, clonidine, levodopa and dopamine agonists) or provoking the release of somatostatin (antimuscarinic agents e.g., atropine).
• Medical history of ongoing clinically symptomatic psychiatric disorders.
• 2nd or 3rd degree atrioventricular-block, prolongation of the QRS complex over 120 milliseconds, prolongation of the QTc interval over 450 milliseconds, or any other clinically significant abnormal electrocardiogram results at the V2 pre-dose electrocardiogram (ECG) as judged by the investigator.
• Previous participation in this trial. Participation is defined as signed informed consent.
• Participation in any clinical trial of an approved or non-approved investigational medicinal product within 30 days before screening.
• Known or suspected hypersensitivity to trial product(s) or related products;
• Any disorder, which in the investigator's opinion might jeopardize subject's safety or compliance with the protocol.
• Concomitant treatment with any drugs that might prolong QT/QTc Note: A subject who receives such treatment will not be a candidate for this study, if his/her condition does not allow for a treatment-free period of at least 5 elimination half-lives of the drug that might prolong QT/QTc before the GHST;
• Elevation of laboratory parameters indicating hepatic or renal dysfunction or damage (aspartate amino transferase (AST), alkaline phosphatase (ALT), gamma-glutamyl transferase (GGT) > 2.5 x upper limit of normal (ULN); creatinine or bilirubin > 1.5x ULN);
• Current active malignancy other than non-melanoma skin cancer;
• Female of child-bearing potential and not using an adequate contraceptive method (adequate contraceptive measures as required by local regulation or practice).
• Male of reproductive age who or whose partner(s) is not using an adequate contraceptive method (adequate contraceptive measures as required by local regulation or practice).
• Lack of ability or willingness to give informed consent by the subject and/or his/her legal representative;
• Anticipated non-availability for trial visits/procedures.
Drug: Macimorelin, Diagnostic Test: Arginine, Diagnostic Test: Clonidine
Growth Hormone Deficiency
childhood-onset growth hormone deficiency, diagnosis of growth hormone deficiency, diagnosis of childhood-onset growth hormone deficiency, diagnostic test for growth hormone deficiency
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A Phase II Multicenter, Randomized, Double-Blind, 12-Week Treatment, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy, Safety and Tolerability of RO7017773 in Participants Aged 15 to 45 Years with Autism Spectrum Disorder (ASD)

Phase II Multicenter, Randomized, Double-Blind, 12-Week Treatment, 3-Arm, Parallel-Group, Placebo-Controlled Study

Suma Jacob
All
15 Years to 45 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04299464
STUDY00010423
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Inclusion Criteria
• Male and female participants with Autism Spectrum Disorder according to Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5)
• Wechsler Abbreviated Scale of Intelligence (WASI-II) >/= 50 at screening or within the last 12 months prior to screening
• ASD or Autism diagnosis confirmed by Autism Diagnostic Observation Schedule (ADOS-2)
• Body mass index within the range of 18.5 to 40 kg/m2
• Female Participants: is eligible if she is not pregnant, not breastfeeding, and women of childbearing potential (WOCBP), who agree to remain abstinent or use contraceptive methods that result in a failure rate of < 1% per year during the treatment period and for at least 28 days after the last dose of study drug
• Language, hearing, and vision compatible with the study measurements as judged by the Investigator
• Allowed existing treatment regimens should be stable for 8 weeks prior to screening. Investigator expects stability of these treatments and behavioral interventions for the duration of the study
• In the Investigator's opinion, able to participate and deemed appropriate for participation in the study, capable of following the study SoA and able to comply with the study restrictions
• In the Investigator's opinion, participation in the study or discontinuation of prohibited medication will not pose undue risks Exclusion Criteria Neurologic/Psychiatric Conditions:
• Non-verbal individuals
• Presence of chromosome 15q11.2 q13.1 duplication syndrome (Dup15q syndrome), known "syndromic" forms of ASD (confirmed per genetic results available at screening): fragile X syndrome, Prader Willi syndrome, Rett's syndrome, tuberous sclerosis, and Angelman syndrome, as well as genetic alterations strongly associated with ASD per genetic results available at screening affecting the following genes: CHD8, ANDP, SHANK3
• Medical history of alcohol and/or substance abuse/dependence in the last 12 months or positive test for drugs of abuse at screening
• Initiation of a major change in psychosocial intervention within 6 weeks prior to screening. Minor changes in ongoing treatment are not considered major changes
• Clinically significant psychiatric and/or neurological disorder that may interfere with the safety or efficacy endpoints
• Risk of suicidal behavior in the opinion of a certified clinician or as evidenced by a "yes" to questions 4 and/or 5 of Columbia-Suicide-Severity Rating Scale (C-SSRS) taken at screening and baseline with respect to the last 12 months, or any suicide attempt in the past 5 years
• Unstable epilepsy/seizure disorder within the past 6 months or changes in anticonvulsive therapy within the last 6 months Other Conditions:
• Medical history of malignancy if not considered cured or if occurred within the last 3 years with the exception of fully excised non-melanoma skin cancers or in-situ carcinoma of the cervix that has been successfully treated
• Concomitant disease, condition or treatment which would either interfere with the conduct of the study or pose an unacceptable risk to the participant in the opinion of the Investigator Prior/Concomitant Therapy
• Use of prohibited medications or herbal remedies within 6 weeks or 5 half-lives (t1/2) prior to randomization Prior/Concurrent Clinical Study Experience:
• Donation or loss of blood over 500 mL in adults and 250 mL in adolescents within 3 months prior to randomization
• Participation in an investigational drug study within 1 month or 5 times the t1/2 of the investigational molecule prior to randomization or participation in a study testing an investigational medical device within 1 month prior to randomization or if the device is still active Diagnostic Assessments
• Confirmed clinically significant abnormality in hematological, chemistry or coagulation laboratory parameters
• Positive test result at screening for hepatitis B surface antigen, hepatitis C virus (HCV, untreated), or human immunodeficiency virus (HIV)-1 and -2. HCV participants who have been successfully treated and who test negative for HCV RNA, may be considered eligible for entry into the study Other Exculsions:
• Uncorrected hypokalemia or hypomagnesaemia
Drug: Placebo, Drug: RO7017773
Autism Spectrum Disorder (ASD)
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MT2020-35 - COG AAML1831 - A Phase 3 Randomized Trial for Patients With De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations

The overall goal of this study is to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, and to find out what effects, good and/or bad, the drug gilteritinib has when given with chemotherapy to children and young adults with newly diagnosed AML and the FLT3/ITD mutation or non-ITD FLT3 activating mutations.

Peter Gordon
All
up to 22 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04293562
STUDY00010751
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Inclusion Criteria:

• All patients must be enrolled on APEC14B1 and consented to Eligibility Screening (Part A) prior to enrollment and treatment on AAML1831. Submission of diagnostic specimens must be done according to the Manual of Procedures
• Patients must be less than 22 years of age at the time of study enrollment
• Patient must be newly diagnosed with de novo AML according to the 2016 World Health Organization (WHO) classification with or without extramedullary disease
• Patient must have 1 of the following:
• >= 20% bone marrow blasts (obtained within 14 days prior to enrollment)
• In cases where extensive fibrosis may result in a dry tap, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy
• < 20% bone marrow blasts with one or more of the genetic abnormalities associated with childhood/young adult AML as provided in the protocol (sample obtained within 14 days prior to enrollment)
• A complete blood count (CBC) documenting the presence of at least 1,000/uL (i.e., a white blood cell [WBC] count >= 10,000/uL with >= 10% blasts or a WBC count of >= 5,000/uL with >= 20% blasts) circulating leukemic cells (blasts) if a bone marrow aspirate or biopsy cannot be performed (performed within 7 days prior to enrollment)
• ARM C: Patient must be >= 2 years of age at the time of Late Callback
• ARM C: Patient must have FLT3/ITD allelic ratio > 0.1 as reported by Molecular Oncology
• ARM C: Patient does not have any congenital long QT syndrome or congenital heart block
• ARM C: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
• ARM C: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
• ARM C: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
• ARM D: Patient must be >= 2 years of age at the time of Late Callback
• ARM D: Patient must have one of the clinically relevant non-ITD FLT3 activating mutations as reported by Foundation Medicine
• ARM D: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
• ARM D: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
• ARM D: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
• NEUROPSYCHOLOGICAL TESTING: Patient must be enrolled on Arm A or Arm B. Patients who transfer to Arm C or Arm D are not eligible
• NEUROPSYCHOLOGICAL TESTING: Patient must be 5 years or older at the time of enrollment
• NEUROPSYCHOLOGICAL TESTING: English-, French- or Spanish-speaking
• NEUROPSYCHOLOGICAL TESTING: No known history of neurodevelopmental disorder prior to diagnosis of AML (e.g., Down syndrome, fragile X, William syndrome, mental retardation)
• NEUROPSYCHOLOGICAL TESTING: No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli
• All patients and/or their parents or legal guardians must sign a written informed consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.
Exclusion Criteria:

• Fanconi anemia
• Shwachman Diamond syndrome
• Patients with constitutional trisomy 21 or with constitutional mosaicism of trisomy 21
• Telomere disorders
• Germline predispositions known, or suspected by the treating physician to increase risk of toxicity with AML therapy
• Any concurrent malignancy
• Juvenile myelomonocytic leukemia (JMML)
• Philadelphia chromosome positive AML
• Mixed phenotype acute leukemia
• Acute promyelocytic leukemia
• Acute myeloid leukemia arising from myelodysplasia
• Therapy-related myeloid neoplasms
• Patients with persistent cardiac dysfunction prior to enrollment, defined as ejection fraction (EF) < 50% (preferred method Biplane Simpson's EF) or if EF unavailable, shortening fraction (SF) < 24%. *Note: if clinically safe and feasible, repeat echocardiogram is strongly advised in order to confirm cardiac dysfunction following clinical stabilization, particularly if occurring in the setting of sepsis or other transient physiologic stressor. If the repeat echocardiogram demonstrates an EF >= 50%, the patient is eligible to enroll and may receive an anthracycline-containing Induction regimen
• Administration of prior anti-cancer therapy except as outlined below:
• Hydroxyurea
• All-trans retinoic acid (ATRA)
• Corticosteroids (any route)
• Intrathecal therapy given at diagnosis
• In particular, strong inducers of CYP3A4 and/or P-glycoprotein (P-gp) should be avoided from the time of enrollment until it is determined whether the patient will receive gilteritinib. Patients receiving gilteritinib will be required to avoid strong CYP3A4 inducers and/or strong P-gp inducers for the duration of the study treatment
• Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
• Lactating females who plan to breastfeed their infants
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
• ARM D: Patient does not have any congenital long QT syndrome or congenital heart block
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation, Drug: Asparaginase, Drug: Asparaginase Erwinia chrysanthemi, Behavioral: Cogstate Assessment Battery, Drug: Cytarabine, Drug: Daunorubicin Hydrochloride, Drug: Dexrazoxane Hydrochloride, Drug: Etoposide, Drug: Gemtuzumab Ozogamicin, Drug: Gilteritinib Fumarate, Drug: Liposome-encapsulated Daunorubicin-Cytarabine, Drug: Methotrexate, Drug: Mitoxantrone Hydrochloride, Drug: Therapeutic Hydrocortisone
Acute Myeloid Leukemia
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A Phase 3, Randomized, Observer-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Immunogenicity of mRNA-1647 Cytomegalovirus (CMV) Vaccine in Healthy Females 16-40 Years of Age (mRNA-1647-P301)

This is a Phase 3, randomized, observer-blinded, placebo-controlled study in healthy participants 16 to 40 years of age. The primary efficacy objective is to demonstrate vaccine effect of mRNA-1647 against primary cytomegalovirus (CMV) infection in female participants who are CMV-seronegative at enrollment.

Mark Schleiss
Female
Not specified
Phase 3
This study is also accepting healthy volunteers
NCT05085366
STUDY00013943
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Inclusion Criteria:
We are recruiting female participants 16-40 years old who are not currently pregnant and have BMIs between 15-35 for this study.
Biological: mRNA-1647, Biological: Placebo
Cytomegalovirus Infection, Infectious Diseases, Women's Health
mRNA-1647, Moderna, Cytomegalovirus, CMV, Cytomegalovirus Vaccine, Cytomegalovirus Infections, Cytomegalovirus Congenital, Virus Diseases, Infection Viral, DNA Virus Infections, Messenger RNA, cmv, cytomegalovirus, vaccine
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A Randomized, Parallel-Arm, Active Control, Multicenter Study Assessing the Safety and Efficacy of DEXTENZA for the Treatment of Ocular Pain and Inflammation Following Surgery for Pediatric Cataract

This randomized trial will compare the insertion of a DEXTENZA plug versus the standard prednisolone acetate suspension in the form of an eye drop to treat ocular pain and inflammation following cataract surgery. Its primary objective is to assess the safety of DEXTENZA compared to the control (prednisolone acetate) in children under the age of 6 years who are undergoing cataract surgery.

Raymond Areaux
Phase III
This study is NOT accepting healthy volunteers
NCT04539548
STUDY00010045
Cataract
Dextenza, dexamethasone ophthalmic insert
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An Open-Label Long-term Follow-up Study to Evaluate the Effects of Sotatercept When Added to Background Pulmonary Arterial Hypertension (PAH) Therapy for the Treatment of PAH

This Phase 3 study is being conducted to assess the long-term safety, tolerability, and efficacy of sotatercept in PAH. Long-term followup of patients receiving sotatercept is important to understand the maintenance and durability of treatment effect (especially in the presence of background PAH therapy) and to provide greater opportunity for pharmacovigilance following sotatercept treatment in the selected patient populations. This LTFU study is supported by data from the PULSAR study (Phase 2, NCT03496207), in which treatment with sotatercept resulted in hemodynamic and functional improvements in the study participants, including those receiving maximal PAH therapy with double/triple drug combinations and intravenous prostacyclin.

Thenappan Thenappan
Phase III
This study is NOT accepting healthy volunteers
NCT04796337
STUDY00016119
PAH
Clinics and Surgery Center (CSC)
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SPR001-205 A Phase 2 Study to Evaluate the Safety, Pharmacokinetics,&#13;&#10;and Exploratory Pharmacodynamics of SPR001 (Tildacerfont) in Children&#13;&#10;Aged 6 to 17 Years with Congenital Adrenal Hyperplasia

This is a Phase 2, open-label, multiple-dose study designed to provide safety, PK, and exploratory PD data for tildacerfont in male and female children aged 6 to 17 years with CAH. The study will last approximately 10 weeks including Screening. After the 14-day Treatment Period, there will be a Safety Follow-up Visit 30 days after the last dose.

Kyriakie Sarafoglou
Phase II
This study is NOT accepting healthy volunteers
NCT05128942
STUDY00014680
21-OHD, Congenital Adrenal Hyperplasia, Rare Diseases
Adrenal Disorder, CAH, Congenital Adrenal Hyperplasia, Pediatric
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A Phase 2, multicenter, open-label, non-randomized, proof-of-concept study evaluating the efficacy, safety, and tolerability of BIVV020 in adults with chronic inflammatory demyelinating polyneuropathy (CIDP)

The purpose of the study is to see if the drug (BIVV020) works to improve symptoms in three populations of adults who have chronic inflammatory demyelinating polyneuropathy (CIDP): • Participants currently on the standard of care (SOC) treatment. • Participants treated previously with the SOC but with no meaningful improvement. • Participants that have not been treated with the SOC. Additional purposes of the study are to find out how safe and tolerable the drug is.

Jeff Allen
18 Years and over
Phase II
This study is NOT accepting healthy volunteers
NCT04658472
STUDY00014727
Chronic Inflammatory Demyelinating Polyradiculoneuropathy, Rare Diseases
CIDP, Clinics and Surgery Center (CSC)
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A 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease (TARGET-IBD)

This is a 5-year, longitudinal, observational study of adult and pediatric patients (age 2 and above) undergoing IBD therapy designed to specifically address important clinical questions that remain incompletely answered from registration trials. Patients being prescribed medical therapy for IBD outside of a clinical trial will be eligible for enrollment. Treatment algorithms will follow each site’s local standard of care and no specific treatments, assessments, and or laboratory tests will be dictated by enrollment in TARGET-IBD. Enrolled patients will consent to the possibility of up to 3 years of retrospective, redacted medical record collection as well as prospective collection for up to 5 years. Medical records will include but will not be limited to: records of hospitalizations, laboratory reports, clinic notes, telephone contact reports, medication lists, reasons for medication initiation and/or discontinuation, endoscopy reports, biopsy results, and imaging results. Patients will also be asked to provide a blood sample for biomarker, anti-drug antibody, and DNA assays and complete patient reported outcome (PRO) surveys, although participation in these two portions of the study is not mandatory for study participation. Consent for linkage of patient health information (PHI) to external healthcare databases (such as patient support programs) will also be requested as an optional portion of the study.

Boris Sudel
All
2 Years and over
NA
This study is NOT accepting healthy volunteers
NCT03251118
STUDY00016242
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Inclusion Criteria:

• Adults and children (age 2 or older) with a diagnosis of Crohn's disease (CD), Ulcerative colitis (UC), or Indeterminate colitis (IBDU) having been prescribed any IBD treatment (initial or subsequent) outside of a clinical trial.
• Have plans for future visits at the site for continued management of IBD.
Exclusion Criteria:

• Inability to provide written informed consent/assent.
• Being enrolled in any interventional study or trial for IBD treatment. Note: Patient may be enrolled in other registries or studies where IBD treatment outcomes are observed and/or reported (such as center-based registries).
• Prior total abdominal colectomy for UC or IBDU.
Inflammatory Bowel Diseases, Crohn's Disease, Ulcerative Colitis, Indeterminate Colitis
Crohn's Disease, Ulcerative colitis, Indeterminate colitis, Digestive System Diseases
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