Phase 2 N=14 Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
Fanconi Anemia
Primary: Number of Participants Experiencing Graft Failure — 1; 0; 0; 0 Participants
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=45 Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
Aplastic Anemia · Leukemia · Myelodysplastic Syndrome
Primary: Successful Neutrophil Engraftment — 44; 1 Participants
Phase 2 N=31 Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
Fanconi Anemia
Primary: Number of Participants Experiencing Graft Failure — 0; 0; 0 Participants
Phase 2 N=1 Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
Fanconi Anemia
Primary: Number of Participants Who Safely Received AMD3100 Used in Combination With Standard Dose G-CSF — 1 participants
N/A N=21 Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
Fanconi Anemia
Primary: Incidence & Quality of Engraftment & Hematopoietic Reconstitution — 19 participants
Phase 2 N=48 Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
Fanconi Anemia · Squamous Cell Carcinoma
Primary: Number of Participants With at Least 20% Reduction of Buccal Micronuclei — 13 Participants
Phase 2 N=6 Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia
Acute Myeloid Leukemia in Remission · de Novo Myelodysplastic Syndrome · Fanconi Anemia
Primary: Number of Patients Who Engraft at Each Dose of TBI Used — 5; 1 Participants
Phase 2 N=14 Pilot Study of Metformin for Patients With Fanconi Anemia
Fanconi Anemia
Primary: Hematologic Response — 4 Participants
N/A N=61 Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Phase 2 N=11 Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia
Anemia, Aplastic · Red-Cell Aplasia, Pure · Anemia, Diamond-Blackfan
Primary: Response to Rituximab — 0; 2; 9 participants
Phase 3 N=36 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Dyskeratosis Congenita · Aplastic Anemia
Primary: Neutrophil Engraftment — 15; 20 Participants
N/A N=9 Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia
Aplastic Anemia
Primary: Number of Patients With Engraftment Response — 9 Participants
Phase 3 N=83 TBI Dose De-escalation for Fanconi Anemia
Fanconi Anemia
Primary: Number of Participant With Neutrophil Recovery — 78 Participants
Phase 2 N=10 MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe Combined Immunodeficiency
Primary: Number of Participants With Adverse Events — 10; 9; 0; 10 participants
Phase 2 N=97 Fludarabine-based Conditioning for Severe Aplastic Anemia (BMT CTN 0301)
Anemia, Aplastic
Primary: Disease-free Survival (DFS) — 6; 3; 9; 4 participants
Phase 2 N=31 Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome
Myelodysplastic Syndrome (MDS) With Refractory Anemia (RA) · Severe Aplastic Anemia (SAA)
Primary: Number of Participants Who Engrafted by Day 42 — 30 Participants
Phase 2 N=47 Alemtuzumab to Treat Severe Aplastic Anemia
Severe Aplastic Anemia, Refractory · Severe Aplastic Anemia, Relapse
Primary: Number of Participants With Hematological Response at 6 Months — 0; 18 Participants
Phase 2 N=12 Interferon Gamma-1b in Friedreich Ataxia (FRDA)
Friedreich Ataxia
Primary: Change in Whole Blood Frataxin Levels — -1.5 percentage of baseline frataxin level — p=0.027
Phase 2 N=1 A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
Aplastic Anemia · Neutropenia · Pancytopenia
Primary: Response Rate at 6 Months — 1; 0; 0 participants
Phase 2 N=12 ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
Immunologic Deficiency Syndromes
Primary: Survival — 12 Participants
Phase 2 N=27 Danazol for Genetic Bone Marrow and Lung Disorders
Aplastic Anemia
Primary: Number of Patients Having Attenuation of Accelerated Telomere Attrition — 12 Participants
N/A N=4 Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Anemia, Aplastic · Amegakaryocytic Thrombocytopenia · Diamond-Blackfan Anemia
Primary: Treatment Failures
Phase 2 N=38 HSCT for High Risk Inherited Inborn Errors
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Phase 3 N=361 Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer
Chronic Myeloproliferative Disorders · Leukemia · Lymphoma
Primary: CR Rate — 42; 55; 134; 18 Participants
Phase 2 N=12 Use of ACTIMMUNE in Patients With ADO2
Autosomal Dominant Osteopetrosis Type 2
Primary: Changes in Bone Resorption Markers From Baseline to 14 Weeks. — 2.2; -2.1 percentage of change
Phase 2 N=15 Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Anemia, Diamond-Blackfan
Primary: Participants That Responded to Eltrombopag — 1; 14 Participants
Phase 2 N=2 Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
Sickle Cell Anemia · Severe Aplastic Anemia · Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Number of Patients With Neutrophil Engraftment — 2 participants
Phase 2 N=282 Clofarabine, Idarubicin, and Cytarabine (CIA) Versus Fludarabine, Idarubicin, and Cytarabine (FLAI) in Acute Myelogenous Leukemia (AML) and High-Risk Myelodysplastic Syndrome
Leukemia
Primary: Maximum Tolerated Dose (MTD) of Clofarabine, Idarubicin, and Cytarabine — 15 mg/m^2
Phase 2 N=34 Eltrombopag for Moderate Aplastic Anemia
Moderate Aplastic Anemia · Unilineage Bone Marrow Failure Disorders
Primary: Proportion of Drug Responders — 17 Participants
Phase 2 N=47 Hematopoietic Cell Transplantation for Pediatric Patients With Hematologic Malignancies
Acute Leukaemia · Chronic Disease · Leukemia
Primary: Number of High Risk Pediatric Patients With Successful Sustained Donor Engraftments — 16; 18; 10 participants