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
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=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=50 Stem Cell Transplantation and T-Cell Add-Back to Treat Bone Marrow Malignancies
Hematologic Malignancies · Bone Marrow Transplant Rejection
Primary: The Proportion of Patients Who Develop Full Donor T Cell Chimerism at Day 30 — 44.9 percentage of participants
Phase 2 N=116 Methods to Enhance the Safety and Effectiveness of Stem Cell Transplants
Chronic Myelogenous Leukemia · Acute Myelogenous Leukemia · Chronic Lymphocytic Leukemia
Primary: Survival and Non-relapse Mortality at Day +200 Using the Miltenyi Reagent System — 43; 4 participants
Phase 3 N=78 Prevention of Osteoporosis in Bone Marrow Transplantation (BMT) Patients
Hematological Malignancies
Primary: Percentage Change in Bone Mineral Density From Baseline to 6 and 12 Months — 0.06; -2.61; -3.63; -4.85 Percentage change
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=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=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 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=62 Modified Stem Cell Transplant Procedure to Treat Patients With Blood and Immune System Cancers
Hematologic Neoplasms
Primary: Number of Recipients Who Developed Grades I-IV Acute Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for…
Phase 2 N=3 Allogeneic Hematopoietic Cell Transplantation for Severe Systemic Sclerosis
Systemic Scleroderma · Severe Systemic Sclerosis
Primary: Event-free Survival (EFS) — 1 Participants
Phase 2 N=66 Busulfan, Melphalan, Fludarabine and T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Followed by Post Transplantation Donor Lymphocyte Infusions
Multiple Myeloma
Primary: Proportion of Participants With Relapsed Multiple Myeloma With Progression-free (PFS) — 0.31 Proportion of participants PFS
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=10 Granulocyte Colony Stimulating Factor (G-CSF) for Bone Marrow Transplant (BMT)
Hematologic Diseases · Hematologic Malignancies
Primary: Numbers of Participants With Disease-free Survival. — 10 participants
Phase 2 N=142 Combination Chemotherapy, Bone Marrow Transplant, and Post Transplant Cyclophosphamide for Hematologic Cancer
Chronic Myeloproliferative Disorders · Leukemia · Lymphoma
Primary: Percentage of Participants Who Develop Acute Graft-versus-host Disease (GVHD) — 49; 10 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=4 Bortezomib to Treat Significant Complication of HSCT
Allogeneic Stem Cell Transplantation · Refractory Autoimmune Cytopenia(s)
Primary: Number of Participants With Response — 3; 2; 1 participants
Phase 2 N=129 Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders
Allogeneic Stem Cell Transplant · Leukemia · Non-Hodgkins
Primary: Overall Survival of Transplant Patients — 727.5 Days
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
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
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=40 Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
Congenital Pediatric Disorders
Primary: Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients. — 0.947 probability of overall survival
Phase 3 N=7 Allogeneic Transplantation For Severe Osteopetrosis
Severe Osteopetrosis
Primary: Number of Patients Who Achieved Donor Cell Engraftment — 5; 0 Participants
Phase 2 N=5 Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant
Primary Immunodeficiency · PID
Primary: Safety: Death — 4 Participants
Phase 3 N=551 Peripheral Blood Stem Cell Transplant vs Bone Marrow Transplant in Individuals With Hematologic Cancers (BMT CTN 0201)
Leukemia · Myeloproliferative Disorders · Myelodysplastic-Myeloproliferative Diseases
Primary: Two-year Overall Survival — 46; 51 percentage of patients
N/A N=3 Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
Lysosomal Storage Disease · Peroxisomal Disorder
Primary: Donor (Allogeneic) Hematopoietic Engraftment — 1 participants
Phase 2 N=89 Chemotherapy and Unrelated Donor Stem Cell Transplantation for Patients With Cancers of the Blood and Immune System
Myelodysplastic Syndrome · Hodgkin's Lymphoma · Non-Hodgkin's Disease
Primary: Percentage of Participants With Grade II-IV Acute Graft Versus Host Disease (GVHD) — 42; 38 percentage of participants
Phase 2 N=31 Stem Cell Transplantation for Patients With Cancers of the Blood
CML (Chronic Myelogenous Leukemia) · CLL (Chronic Lymphocytic Leukemia) · AML (Acute Myelogenous Leukemia)
Primary: To Determine if Selective T Cell Depletion Using the Photodepletion Procedure Can Substantially Reduce the Rate of Severe Acute GVHD (Grade III/IV) After Matched Sibling…