42 completed, 15 unknown, 14 terminated, 10 recruiting. 98 Phase 1, 66 Phase 2.
Follow Anemia, Aplastic: new trials by email Literature Review of Anemia, Aplastic trials Research Report on Anemia, Aplastic
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 |
|---|---|---|---|---|---|---|
| Prenatal Transplantation for Fetuses With Fanconi Anemia IUHSCT for FA-affected fetuses - Fanconi Anemia, Anemia, Hypoplastic, Congenital | Phase 1 / Phase 2 | Not yet recruiting | Agnieszka Czechowicz | 12 | 2028-01-01 | 2026-06-16 |
| Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms pacritinib - Myelodysplastic Syndromes | Phase 1 / Phase 2 | Recruiting | National Cancer Institute (NCI) | 160 | 2026-11-08 | 2026-11-03 |
| Safety and Tolerability of Metformin and Naproxen for Cancer Prevention in Fanconi Anemia Metformin, Naproxen Sodium - Fanconi Anemia, Head and Neck Cancer | Phase 1 | Recruiting | Rockefeller University | 80 | 2026-10-14 | 2026-10-28 |
| Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI Sirolimus, Ruxolitinib, MMF - Acute Leukemia, Myelodysplastic Syndromes | Phase 1 / Phase 2 | Not yet recruiting | Peking University People's Hospital | 40 | 2026-10-01 | 2026-09-09 |
| 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 | Not yet recruiting | Dren Bio | 60 | 2026-08-01 | 2026-07-24 |
| Phase I/II Study of Anti-CD38 Monoclonal Antibody in Refractory Severe Aplastic Anemia Isatuximab - Aplastic Anemia | Phase 1 / Phase 2 | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 32 | 2026-06-30 | 2026-09-21 |
| Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia APR-2020 - RPS19 Deficient Diamond-Blackfan Anemia | Phase 1 | Recruiting | Apriligen, Inc. | 4 | 2026-04-16 | 2026-07-21 |
| Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia Golidocitinib Capsules - Severe Aplastic Anemia, Refractory Aplastic Anemia | Phase 1 / Phase 2 | Not yet recruiting | Institute of Hematology & Blood Diseases Hospital, China | 42 | 2025-12-15 | 2025-12-22 |
| The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia Daratumumab - Aplastic Anemia, Refractory | Phase 1 / Phase 2 | Not yet recruiting | Institute of Hematology & Blood Diseases Hospital, China | 37 | 2025-12-10 | 2025-12-17 |
| CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia RD13-02 - Severe Aplastic Anemia, Relapse | Phase 1 | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 15 | 2025-06-19 | 2025-07-24 |
| Study of CM313(SC) Injection in Subjects With Relapsed/Refractory Aplastic Anemia CM313(SC) injection - Aplastic Anemia | Phase 1 / Phase 2 | Not yet recruiting | Keymed Biosciences Co.Ltd | 125 | 2025-03-01 | 2025-02-18 |
| A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia Anti-Thymocyte Globulin, Biospecimen Collection, Bone Marrow Aspirate - Recurrent Severe Aplastic Anemia, Refractory Severe Aplastic Anemia | Phase 1 | Recruiting | City of Hope Medical Center | 6 | 2024-11-15 | 2026-01-05 |
| Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC) Afatinib - Fanconi Anemia, Head and Neck Squamous Cell Carcinoma | Phase 1 / Phase 2 | Recruiting | Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | 25 | 2024-11-08 | 2026-05-27 |
| REscuing Bone Marrow Function in Patients with AplaStic AnaEmia and Bone Marrow FaiLure Post AllogEneiC Transplantation 2 Ruxolitinib (JAKAVI®), Eltrombopag (Revolade®) - Poor Graft Function, Aplastic Anemia Idiopathic | Phase 1 / Phase 2 | Not yet recruiting | Melbourne Health | 20 | 2024-10-30 | 2024-09-23 |
| Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to Calcineurin Inhibitors Sirolimus, Abatacept, MMF - Acute Leukemia, Myelodysplastic Syndromes | Phase 1 / Phase 2 | Unknown | Peking University People's Hospital | 6 | 2024-03-01 | 2024-03-21 |
| JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure Ruxolitinib - Severe Aplastic Anemia, Single Lineage Cytopenias, T-LGL | Phase 1 / Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 13 | 2024-02-20 | 2026-10-14 |
| Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia Bitopertin - Steroid-refractory Diamond-Blackfan Anemia (DBA) | Phase 1 / Phase 2 | Completed | National Heart, Lung, and Blood Institute (NHLBI) | 15 | 2023-07-25 | 2026-06-30 |
| A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia FP-045 - Fanconi Anemia | Phase 1 / Phase 2 | Withdrawn | Foresee Pharmaceuticals Co., Ltd. | 2023-06-30 | 2025-03-06 | |
| Efficacy of Daratumumab to Overcome Platelet Transfusion Refractoriness in Patients with Aplastic Anemia Daratumumab - Aplastic Anemia, Platelet Transfusion Refractoriness | Phase 1 | Completed | Institute of Hematology & Blood Diseases Hospital, China | 10 | 2023-04-17 | 2025-01-03 |
| Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA 3D hypoxia-preconditioned UC-MSC group, 2D UC-MSC group - Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Refractory | Phase 1 / Phase 2 | Enrolling by invitation | Chinese PLA General Hospital | 40 | 2022-08-01 | 2026-06-23 |
Showing 20 of 98. Sign in to see all of them.
Fanconi Anemia (30) · Myelodysplastic Syndromes (19) · Anemia, Diamond-Blackfan (17) · Bone Marrow Failure Disorders (11) · Anemia, Sickle Cell (10) · Leukemia, Myeloid, Acute (10) · Lymphoma, Non-Hodgkin (10) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (10) · Primary Immunodeficiency Diseases (8) · Hematologic Neoplasms (7) · Hemoglobinopathies (7) · Hodgkin Disease (6) · Leukemia (6) · Myeloproliferative Disorders (6)
Hemic and Lymphatic Diseases (98) · Bone Marrow Diseases (97) · Anemia (94) · Hematologic Diseases (94) · Bone Marrow Failure Disorders (87) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (48) · Genetic Diseases, Inborn (46) · Congenital Bone Marrow Failure Syndromes (40) · Anemia, Hypoplastic, Congenital (39) · Nutritional and Metabolic Diseases (36) · Metabolic Diseases (35) · DNA Repair-Deficiency Disorders (31) · Immune System Diseases (25) · Neoplasms (20)
Cyclophosphamide (16) · fludarabine (13) · Whole-Body Irradiation (10) · Cyclosporine (9) · fludarabine phosphate (9) · Filgrastim (8) · Antilymphocyte Serum (7) · Granulocyte Colony-Stimulating Factor (7) · Alemtuzumab (6) · Mycophenolic Acid (5) · Peripheral Blood Stem Cell Transplantation (5) · Melphalan (4) · Methylprednisolone (4) · Sirolimus (4) · plerixafor (4)
National Heart, Lung, and Blood Institute (NHLBI) (11) · Fred Hutchinson Cancer Center (5) · Institute of Hematology & Blood Diseases Hospital, China (5) · Bellicum Pharmaceuticals (4) · Baylor College of Medicine (3) · Children's Hospital Medical Center, Cincinnati (3) · Boston Children's Hospital (2) · Diane George (2) · Joanne Kurtzberg, MD (2) · M.D. Anderson Cancer Center (2) · Masonic Cancer Center, University of Minnesota (2) · Northwell Health (2) · Peking University People's Hospital (2) · Sierra Oncology LLC - a GSK company (2) · Talaris Therapeutics Inc. (2)
United States (63) · China (11) · United Kingdom (6) · Brazil (3) · Germany (3) · South Korea (3) · Spain (3) · France (2) · Italy (2) · Australia (1) · Austria (1) · Norway (1) · Russia (1) · Singapore (1)
Adrianna Vlachos, MD (3) · Danielle M Townsley, M.D. (2) · David J Young, M.D. (2) · Diane George, MD (2) · Hans-Peter Kiem (2) · Joanne Kurtzberg, MD (2) · John E. Wagner, Jr. (2) · Neal S Young, M.D. (2) · Agata Smogorzewska, MD, PhD (1) · Ana Beatriz Hortense, MD (1) · Ann Woolfrey (1) · Baoyong Yan, Doctor (1)
PMID 25824692, PMID 34724566, PMID 21742281, PMID 22055111, PMID 22546497, PMID 23731760, PMID 24296492, PMID 25185712, PMID 25310303, PMID 26354756, PMID 26589752, PMID 26685770, PMID 27192671, PMID 29419434, PMID 29674506, PMID 29958797, PMID 31501599, PMID 33025707, PMID 33410523, PMID 33910334, PMID 34525188, PMID 34665631, PMID 34751952, PMID 35371427, PMID 36378548, PMID 36579443, PMID 37120671, PMID 37142543, PMID 38467031, PMID 39642902, PMID 39820512, PMID 40331918, PMID 40696207, PMID 40859225, PMID 41742894, PMID 42583025
R01HL081499 (2), 2014 (1), 2023 (1), P30CA015704 (1), P30CA033572 (1), P30CA036727 (1), U01HL069294 (1), U24CA076518 (1), U54RR019397 (1)