7 completed, 4 recruiting, 1 active not recruiting, 1 suspended. 10 Phase 2, 5 Phase 1, 2 Early Phase 1, 2 Phase 3.
Follow Bone Marrow Failure Syndromes: new trials by email Literature Review of Bone Marrow Failure Syndromes trials Research Report on Bone Marrow Failure Syndromes
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 |
|---|---|---|---|---|---|---|
| Methadone as an Alternative Treatment for Children Underdoing HSCT PCA plus methadone (Experimental), PCA Alone (SOC) - Leukemia, Aplastic Anemia | Phase 3 | Suspended | University of Texas Southwestern Medical Center | 60 | 2028-01-01 | 2025-10-23 |
| A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes Dibotatug (DR-01) - Bone Marrow Failure Syndromes, Aplastic Anemias | Phase 1 / Phase 2 | Recruiting | Dren Bio | 60 | 2026-08-14 | 2026-11-04 |
| UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep Fludarabine, Cyclophosphamide, MMF - Acute Leukemia, Acute Myeloid Leukemia | Phase 2 | Completed | Masonic Cancer Center, University of Minnesota | 15 | 2017-05-15 | 2025-07-04 |
| Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders RIC regimen, GVHD prophylaxis regimen - Severe Sickle Cell Disease, Bone Marrow Failure Syndromes | Phase 1 / Phase 2 | Recruiting | Washington University School of Medicine | 29 | 2017-03-20 | 2026-05-29 |
| Investigation of the Genetics of Hematologic Diseases Bone Marrow Failure Syndromes, Erythrocyte Disorder | Recruiting | St. Jude Children's Research Hospital | 1716 | 2016-06-17 | 2026-06-09 | |
| Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases Treosulfan, Busilvex - Primary Immunodeficiencies, Inborn Errors of Metabolism | Phase 2 | Completed | medac GmbH | 106 | 2015-04-01 | 2025-12-08 |
| Fludarabine Based RIC for Bone Marrow Failure Syndromes MRD-BMT with Fludarabine-based RIC for Acquired AA, MRD-BMT with Fludarabine-based RIC for iBMF with trilineage aplasia, MRD-BMT with Fludarabine-based RIC for iBMF without trilineage aplasia - Bone Marrow Failure Syndromes | Early Phase 1 | Active not recruiting | Children's Hospital of Philadelphia | 25 | 2015-04-01 | 2026-05-28 |
| Memory T-cell Infusion to Improve Immunity After TCR-alpha/Beta Depleted Hematopoietic Stem Cell Transplantation CD45RA-depleted peripheral blood mononuclear cells - Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute | Phase 1 / Phase 2 | Completed | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | 30 | 2014-08-01 | 2016-04-22 |
| Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia Bone marrow transplant, Thymoglobulin, Fludarabine - Severe Aplastic Anemia, Bone Marrow Failure Syndromes | Phase 2 | Completed | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | 18 | 2014-08-01 | 2023-03-10 |
| Nandrolone Decanoate in the Treatment of Telomeropathies Nandrolone Decanoate - Aplastic Anemia, Bone Marrow Failure Syndromes | Phase 1 / Phase 2 | Completed | University of Sao Paulo | 20 | 2014-02-01 | 2023-08-31 |
| Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs) - Leukemia, Inborn Errors of Metabolism | Early Phase 1 | Recruiting | Children's Hospital of Philadelphia | 100 | 2013-12-01 | 2026-02-20 |
| Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases CliniMACs device - Bone Marrow Failure Syndromes, Immunodeficiencies | Phase 2 | Completed | Children's Hospital of Philadelphia | 2 | 2011-11-01 | 2018-04-11 |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies Enriched Hematopoetic Stem Cell Infusion - Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies | Phase 1 / Phase 2 | Withdrawn | Talaris Therapeutics Inc. | 2011-05-01 | 2023-03-03 | |
| Identifying Characteristics of Bone Marrow Failure Syndromes Bone Marrow Failure Syndromes, Anemia, Aplastic | Unknown | Office of Rare Diseases (ORD) | 450 | 2006-04-01 | 2010-04-21 | |
| Comparison of Cy-Atg Vs Cy-Flu-Atg for the Conditioning Therapy in Allo-HCT Cyclophosphamide-fludarabine-anti thymocyte globulin - Bone Marrow Failure Syndromes | Phase 3 | Completed | Cooperative Study Group A for Hematology | 82 | 2003-03-01 | 2011-02-17 |
| Unrelated Donor Transplant for Malignant and Non-Malignant Disorders UDAlloSCT, Therapy - Leukemia, Lymphoma | Phase 2 | Terminated | Columbia University | 22 | 2002-11-01 | 2015-04-15 |
Bone Marrow Failure Disorders (16) · Anemia, Aplastic (5) · Leukemia (4) · Anemia, Sickle Cell (3) · Immunologic Deficiency Syndromes (3) · Myelodysplastic Syndromes (3) · Anemia, Diamond-Blackfan (2) · Graft vs Host Disease (2) · Hemoglobinopathies (2) · Leukemia, Myeloid, Acute (2) · Leukocyte Disorders (2) · Metabolism, Inborn Errors (2) · Blood Coagulation Disorders (1) · Burkitt Lymphoma (1) · Dendritic Cell Sarcoma, Interdigitating (1)
Bone Marrow Diseases (16) · Hemic and Lymphatic Diseases (16) · Hematologic Diseases (15) · Anemia (8) · Immune System Diseases (6) · Neoplasms by Histologic Type (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Neoplasms (5) · Anemia, Hemolytic (4) · Immunoproliferative Disorders (4) · Lymphatic Diseases (4) · Lymphoproliferative Disorders (4) · Nutritional and Metabolic Diseases (4) · Pathological Conditions, Signs and Symptoms (4)
Cyclophosphamide (3) · Mycophenolic Acid (2) · fludarabine (2) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Hydromorphone (1) · Mesna (1) · Methadone (1) · Morphine (1) · Nandrolone Decanoate (1) · Passive Cutaneous Anaphylaxis (1) · Sirolimus (1) · Tacrolimus (1) · Therapeutics (1)
Children's Hospital of Philadelphia (3) · Columbia University (1) · Cooperative Study Group A for Hematology (1) · Dren Bio (1) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (1) · Masonic Cancer Center, University of Minnesota (1) · Office of Rare Diseases (ORD) (1) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (1) · St. Jude Children's Research Hospital (1) · Talaris Therapeutics Inc. (1) · University of Sao Paulo (1) · University of Texas Southwestern Medical Center (1) · Washington University School of Medicine (1) · medac GmbH (1)
United States (12) · Brazil (1) · Czechia (1) · Germany (1) · Italy (1) · Poland (1) · Russia (1) · South Korea (1)
Timothy Olson, MD, PhD (2) · Amy DeZern, MD (1) · Ana Beatriz Hortense, MD (1) · Jaroslaw P. Maciejewski, MD, PhD (1) · Karl-Walter Sykora, MD and Prof (1) · Kyoo-Hyung Lee, Doctor (1) · Marcin Wlodarski, MD, PhD (1) · Margaret MacMillan, MD, MSC (1) · Michael Maschan, MD (1) · Mitchell S Cairo, MD (1) · Nancy Bunin, MD (1) · Rodrigo T Calado, MD, PhD (1) · Shalini Shenoy, MD (1)