6 recruiting, 3 completed, 2 active not recruiting, 2 terminated. 13 Phase 2, 3 Phase 1, 1 Not applicable, 1 Phase 3.
Follow Bone Marrow Failure: new trials by email Literature Review of Bone Marrow Failure trials Research Report on Bone Marrow Failure
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs - Bone Marrow Failure, Hemoglobinopathies | Phase 2 | Not yet recruiting | Fred Hutchinson Cancer Center | 40 | 2026-09-01 | 2026-06-22 |
| The HOPE Biobank Resource (BMT CTN 2402 HOPE) Aplastic Anemias, Hemoglobinopathies | Recruiting | Medical College of Wisconsin | 375 | 2026-05-15 | 2026-08-27 | |
| Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT Phase 1 Dose Level 1, Phase 1 Dose Level 2, Phase 1 Dose Level 3 - Leukemia, High Risk Acute Lymphoblastic Leukemia | Phase 1 / Phase 2 | Recruiting | Children's Hospital of Philadelphia | 100 | 2025-03-21 | 2026-04-15 |
| European Rare Blood Disorders Platform (ENROL) Collection of clinical and laboratory data from EHR. - Anemia, Bone Marrow Failure | Recruiting | Hospital Universitari Vall d'Hebron Research Institute | 37090 | 2022-07-01 | 2024-02-09 | |
| Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure EXG34217 - Telomere Shortening, Bone Marrow Failure | Phase 1 | Active not recruiting | Elixirgen Therapeutics, Inc. | 12 | 2021-04-08 | 2026-02-06 |
| Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders Clofarabine, Fludarabine, Busulfan - AML, ALL | Phase 2 | Recruiting | Memorial Sloan Kettering Cancer Center | 31 | 2020-11-20 | 2026-03-13 |
| Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures Alemtuzumab, Fludarabine, Melphalan - Primary Immune Deficiency Disorder, Immune Deficiency Disease | Phase 2 | Recruiting | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | 27 | 2020-02-12 | 2025-11-28 |
| Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant Anti-Thymocyte Globulin, Cyclophosphamide, Cytokine-treated Veto Cells - Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia | Phase 1 / Phase 2 | Active not recruiting | M.D. Anderson Cancer Center | 16 | 2019-08-08 | 2026-06-11 |
| Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU) - Fanconi Anemia, Severe Aplastic Anemia | Phase 2 | Recruiting | Masonic Cancer Center, University of Minnesota | 48 | 2018-11-13 | 2026-01-30 |
| Identification of Autoantibodies and Autoantigens in the Cerebrospinal Fluid of Patients With Spinal Cord Trauma Bone Marrow Failure, Trauma | Withdrawn | University Hospital, Lille | 2018-09-01 | 2019-12-05 | ||
| Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study Red Blood Cell Transfusions (RBCT) - Anemia, Aplastic Anemia | Not applicable | Withdrawn | Emory University | 2017-07-24 | 2018-07-26 | |
| NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia Thymoglobulin, Fludarabine, Cyclophosphamide - Severe Aplastic Anemia, Aplastic Anemia | Phase 2 | Completed | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | 21 | 2016-09-01 | 2022-07-12 |
| Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) Levamisole+cyclosporin A+Glucocorticoids, cyclosporin A+Glucocorticoids, Glucocorticoids - Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria | Phase 2 | Unknown | Institute of Hematology & Blood Diseases Hospital, China | 50 | 2013-01-01 | 2013-01-03 |
| Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria Levamisole+cyclosporin A+Glucocorticoids, cyclosporin A+Glucocorticoids, Glucocorticoids - Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria | Phase 2 | Unknown | Institute of Hematology & Blood Diseases Hospital, China | 120 | 2012-07-01 | 2013-01-03 |
| CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant Full Intensity with TBI, Full Intensity, Reduced Intensity - Leukemia, Lymphoma | Phase 2 | Completed | New York Medical College | 20 | 2011-09-01 | 2018-08-21 |
| CD34+ Stem Cell Infusion to Augment Graft Function CD34+ - Waning Donor Chimerism, Waning Immune Function | Phase 2 | Terminated | Children's Hospital Medical Center, Cincinnati | 23 | 2010-10-01 | 2019-11-22 |
| T-cell Depleted Alternative Donor Transplantation CliniMACS® (T cell depletion) - Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia | Phase 2 | Terminated | Wake Forest University Health Sciences | 53 | 2009-08-01 | 2022-04-22 |
| Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases Fludarabine, Cyclophosphamide, Busulfan - Bone Marrow Failure, Osteopetrosis | Phase 2 / Phase 3 | Completed | Columbia University | 38 | 2002-09-01 | 2024-10-31 |
| Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant CD34+ enriched, T Cell Depleted donor stem cell product - Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia | No longer available | Christopher Dvorak | 2021-05-27 |
Bone Marrow Failure Disorders (19) · Anemia, Aplastic (7) · Hemoglobinopathies (6) · Leukemia (5) · Myelodysplastic Syndromes (5) · Anemia (4) · Immunologic Deficiency Syndromes (4) · Leukemia, Myeloid, Acute (4) · Fanconi Anemia (3) · Hematologic Diseases (3) · Leukemia, Myelogenous, Chronic, BCR-ABL Positive (3) · Lymphoma (3) · Lymphoma, Non-Hodgkin (3) · Myeloproliferative Disorders (3) · Osteopetrosis (3)
Hemic and Lymphatic Diseases (19) · Bone Marrow Diseases (18) · Hematologic Diseases (16) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Genetic Diseases, Inborn (10) · Anemia (9) · Immune System Diseases (9) · Neoplasms (7) · Neoplasms by Histologic Type (7) · Lymphatic Diseases (6) · Metabolic Diseases (6) · Nutritional and Metabolic Diseases (6) · Immunoproliferative Disorders (5) · Lymphoproliferative Disorders (5) · Pathological Conditions, Signs and Symptoms (5)
Cyclophosphamide (6) · fludarabine (6) · Whole-Body Irradiation (5) · Alemtuzumab (4) · Antilymphocyte Serum (4) · Busulfan (4) · fludarabine phosphate (4) · Melphalan (3) · Mycophenolic Acid (3) · Cyclosporine (2) · Glucocorticoids (2) · Levamisole (2) · Tacrolimus (2) · thymoglobulin (2) · Blood Component Transfusion (1)
Institute of Hematology & Blood Diseases Hospital, China (2) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (2) · Children's Hospital Medical Center, Cincinnati (1) · Children's Hospital of Philadelphia (1) · Christopher Dvorak (1) · Columbia University (1) · Elixirgen Therapeutics, Inc. (1) · Emory University (1) · Fred Hutchinson Cancer Center (1) · Hospital Universitari Vall d'Hebron Research Institute (1) · M.D. Anderson Cancer Center (1) · Masonic Cancer Center, University of Minnesota (1) · Medical College of Wisconsin (1) · Memorial Sloan Kettering Cancer Center (1) · New York Medical College (1)
United States (15) · China (2) · Spain (1)
Amy E DeZern, MD (1) · Ana Antun, MD (1) · Andrew Gilman, MD (1) · Christopher Dvorak, MD (1) · Fahed Zairi, MD (1) · Heather J Symons, MD, MHS (1) · James Garvin, MD, PhD (1) · Kasiani Myers, MD (1) · Madhavi Lakkaraja, MD, MPH (1) · Margaret MacMillan, MD, Msc, FRCPC (1) · Maria Cancio, MD (1) · Mitchell S Cairo, MD (1)
PMID 29296761, PMID 29555312, PMID 32343796, PMID 33159275, PMID 37084383, PMID 39998303