Phase 3 N=10 Stem Cell Transplant for Bone Marrow Failure Syndromes
Diamond-Blackfan Anemia · Kostmann's Neutropenia · Shwachman-Diamond Syndrome
Primary: Number of Patients Alive (Survival) at 2 Years — 6 Participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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=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=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=6 Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 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 2 N=30 Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)
Sickle Cell Disease
Primary: Percentage of Participants With Event-Free Survival (EFS) — 76; 69 percentage of participants
Phase 2 N=63 Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome
Myelofibrosis
Primary: Rate of Non-relapse Mortality at 100 Days Post-transplant — 1; 2; 1 Participants
Phase 2 N=46 Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Mucopolysaccharidosis · Hurler Syndrome · Hunter Syndrome
Primary: Number of Patients With Donor Derived Engraftment — 42 Participants
Phase 2 N=202 Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow
Myeloproliferative Disorders · Acute Myelogenous Leukemia · Chronic Myelogenous Leukemia
Primary: Number of Participants Who Experienced Transplant Related Mortality — 5; 4 Participants
Phase 2 N=47 Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
Hemophagocytic Lymphohistiocytosis · Chronic Active Epstein-Barr Virus Infection · Chronic Granulomatous Disease
Primary: Percentage of Participants With Overall Survival (OS) — 80.4; 66.7 percentage of participants
Phase 2 N=55 Bone Marrow Transplant From Partially Matched Donors and Nonmyeloablative Conditioning for Blood Cancers (BMT CTN 0603)
Precursor B-Cell Lymphoblastic Leukemia-Lymphoma · Leukemia, Myeloid, Acute · Burkitt Lymphoma
Primary: Overall Survival at 180 Days From the Time of Transplant — 83.7; 62.0 percentage of participants
Phase 3 N=36 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Dyskeratosis Congenita · Aplastic Anemia
Primary: Neutrophil Engraftment — 15; 20 Participants
Phase 3 N=25 Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome · Acute Myeloid Leukemia/Transient Myeloproliferative Disorder · Adult Acute Myeloid Leukemia in Remission
Primary: Overall Survival — 6; 6 participants
N/A N=62 Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
Accelerated Phase Chronic Myelogenous Leukemia · Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome · Adult Acute Lymphoblastic Leukemia in Remission
Primary: Day 100 TRM — 8.44 percentage of participants
Early Phase 1 N=48 Donor Stem Cell Transplantation for Congenital Immunodeficiencies
Inherited Immune Deficiencies
Primary: Stem Cell Transplant Engraftment — 7; 31; 3; 0 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=95 Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
Sickle Cell Disease
Primary: Percentage of Participants With Two-Year Post-Transplant Event Free Survival (EFS) — 88.0; 79.3 Percentage of Participants
Phase 2 N=123 Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)
Sickle Cell Disease
Primary: Percentage of Participants With Overall Survival (OS) at 2 Years After Biologic Assignment — 89; 94 percentage of 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
N/A N=2 Combined Bone Marrow and Renal Transplantation for Hematologic Disorders With End Stage Renal Disease
Multiple Myeloma · Amyloidosis
Primary: Number of Participants Without Renal Allograft Rejection at 6 Months Post-transplant — 1 Participants
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
Phase 2 N=136 Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia
Immunosuppresion · Thrombocytopenia · Pancytopenia
Primary: Hematologic Response — 38; 20; 3 Participants
Phase 2 N=17 Reduced-Intensity Regimen Before Donor Bone Marrow Transplant in Treating Patients With Myelodysplastic Syndromes
Leukemia · Myelodysplastic Syndromes · Myelodysplastic/Myeloproliferative Neoplasms
Primary: Complete Response Rate — 35.3 percentage of participants
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 2 N=22 Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
Sickle Cell Disease
Primary: Event -Free Survival Rate — 19 Participants
Phase 2 N=15 Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)
Juvenile Myelomonocytic Leukemia
Primary: Determine Probability of Long-term Disease Free Survival in JMML — 67 Percentage of participants
N/A N=29 Calcium Absorption in Patients With Rothmund-Thomson Syndrome
Rothmund-Thomson Syndrome
Primary: Bone Density (Low Areal Bone Mineral Density (aBMD)) — -1.3; -0.9; -2.4; -1.2 z-score
Phase 2 N=48 Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
Myelodysplastic Syndrome · Aplastic Anemia
Primary: Overall Response — 8; 8; 4 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