110 completed, 31 recruiting, 31 unknown, 24 terminated. 232 Phase 2, 66 Phase 1, 14 Phase 3.
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 |
| Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBT Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®) - Aplastic Anaemia | Phase 2 | Not yet recruiting | Anhui Provincial Hospital | 59 | 2027-08-01 | 2026-10-21 |
| CK0801 for Treatment of Aplastic Anemia CK0801 - Aplastic Anemia | Phase 2 | Not yet recruiting | Cellenkos, Inc. | 12 | 2026-12-03 | 2026-10-20 |
| 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 | 2026-11-17 | 2026-11-12 |
| 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 | Recruiting | Dren Bio | 60 | 2026-08-14 | 2026-11-04 |
| A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment Avatrombopag 20 mg Oral Tablet - Aplastic Anemia | Phase 2 / Phase 3 | Not yet recruiting | Swedish Orphan Biovitrum | 26 | 2026-07-31 | 2026-08-26 |
| Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation Intravenous Immunoglobulin, Dexamethasone, Mononuclear Cells - Leukemia, Lymphoma | Phase 2 | Not yet recruiting | Hematology department of the 920th hospital | 60 | 2026-07-03 | 2026-07-14 |
| 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 |
| Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA Romiplostim N01 - Aplastic Anemia | Phase 2 | Withdrawn | Peking Union Medical College Hospital | 2026-06-01 | 2026-06-04 | |
| CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA Cyclosporine (CsA), Thrombopoietin Receptor Agonist, Romiplostim N01 - Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia | Phase 2 | Withdrawn | Peking Union Medical College Hospital | 2026-06-01 | 2026-06-04 | |
| Romiplostim N01 in Chemoradiotherapy-induced Aplastic Anemia Romiplostim N01 - Aplastic Anemia | Phase 2 | Not yet recruiting | Peking Union Medical College Hospital | 43 | 2026-04-01 | 2026-05-07 |
| CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA Cyclosporine (CsA), thrombopoietin receptor agonist (TPO-RA), Romiplostim N01 - Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia | Phase 2 | Not yet recruiting | Peking Union Medical College Hospital | 43 | 2026-04-01 | 2026-04-30 |
| Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA Romiplostim, Recombinant Human Thrombopoietin - MDS (Myelodysplastic Syndrome), Aplastic Anemia (AA) | Phase 2 | Recruiting | The First Affiliated Hospital of Soochow University | 66 | 2026-01-31 | 2026-04-15 |
| The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia Daratumumab - Aplastic Anemia, Refractory | Phase 1 / Phase 2 | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 37 | 2025-12-17 | 2026-11-06 |
| 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 |
| Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy - Aplastic Anemia | Phase 2 | Active not recruiting | Fred Hutchinson Cancer Center | 1 | 2025-09-25 | 2026-05-22 |
| Efficacy and Safety of UCBT With TMI -Based Conditioning Regimen for Adults With Refractory/Relapsed Aplastic Anemia Total Marrow Irradiation, Fludarabine (FLU), Melphalan (Mel) - Aplastic Anemia Idiopathic | Phase 2 | Not yet recruiting | Institute of Hematology & Blood Diseases Hospital, China | 11 | 2025-07-20 | 2025-07-22 |
| Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia Haploidentical donor bone marrow transplant, Unrelated donor bone marrow transplant - Severe Aplastic Anemia | Phase 2 | Recruiting | Medical College of Wisconsin | 60 | 2025-05-12 | 2026-02-17 |
| UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia Romiplostim, Immunosuppressive therapy (IST) - Aplastic Anemia | Phase 2 | Withdrawn | Anjali Sharathkumar | 2025-04-10 | 2026-10-16 |
Showing 20 of 232. Sign in to see all of them.
Myelodysplastic Syndromes (49) · Fanconi Anemia (38) · Anemia, Diamond-Blackfan (32) · Bone Marrow Failure Disorders (29) · Leukemia, Myeloid, Acute (28) · Lymphoma, Non-Hodgkin (25) · Hodgkin Disease (24) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (24) · Leukemia (23) · Multiple Myeloma (23) · Leukemia, Lymphocytic, Chronic, B-Cell (19) · Anemia, Sickle Cell (17) · Myeloproliferative Disorders (17) · Leukemia, Myelogenous, Chronic, BCR-ABL Positive (16)
Hemic and Lymphatic Diseases (232) · Bone Marrow Diseases (230) · Anemia (225) · Hematologic Diseases (221) · Bone Marrow Failure Disorders (203) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (80) · Genetic Diseases, Inborn (71) · Congenital Bone Marrow Failure Syndromes (66) · Anemia, Hypoplastic, Congenital (63) · Immune System Diseases (63) · Neoplasms by Histologic Type (52) · Nutritional and Metabolic Diseases (52) · Pathological Conditions, Signs and Symptoms (52) · Metabolic Diseases (50)
Cyclophosphamide (56) · fludarabine (44) · Cyclosporine (43) · fludarabine phosphate (38) · Antilymphocyte Serum (36) · Whole-Body Irradiation (31) · Mycophenolic Acid (24) · Alemtuzumab (23) · Busulfan (23) · Tacrolimus (23) · thymoglobulin (22) · eltrombopag (18) · Filgrastim (17) · Melphalan (17) · Granulocyte Colony-Stimulating Factor (16)
National Heart, Lung, and Blood Institute (NHLBI) (31) · Institute of Hematology & Blood Diseases Hospital, China (15) · Masonic Cancer Center, University of Minnesota (9) · Novartis Pharmaceuticals (9) · Peking Union Medical College Hospital (9) · Fred Hutchinson Cancer Center (7) · Assistance Publique - Hôpitaux de Paris (5) · M.D. Anderson Cancer Center (5) · Roswell Park Cancer Institute (5) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (5) · Medical College of Wisconsin (4) · Memorial Sloan Kettering Cancer Center (4) · Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University (4) · Asan Medical Center (3) · Baylor College of Medicine (3)
United States (136) · China (34) · South Korea (12) · Italy (7) · Japan (6) · Spain (6) · United Kingdom (6) · France (5) · Germany (5) · Brazil (4) · Russia (3) · Thailand (3) · Hong Kong (2) · Norway (2) · Taiwan (2)
Neal S Young, M.D. (6) · Danielle M Townsley, M.D. (5) · Richard W Childs, M.D. (4) · Adrianna Vlachos, MD (3) · Cynthia E Dunbar, M.D. (3) · Margaret MacMillan, MD (3) · Usama Gergis, MD (3) · Andrew M Yeager, MD (2) · Bhavisha A Patel, M.D. (2) · Bhavisha Patel, M.D. (2) · David E Avigan, MD (2) · David F McDermott, MD (2) · David J Young, M.D. (2)
PMID 34724566, PMID 29958797, PMID 29674506, PMID 20685256, PMID 22055111, PMID 22067384, PMID 25310303, PMID 25824692, PMID 30992268, PMID 32343796, PMID 40593138, PMID 1090310, PMID 12622583, PMID 18723429, PMID 19117511, PMID 19181786, PMID 20383216, PMID 21606164, PMID 21742281, PMID 21874061, PMID 22517900, PMID 22544699, PMID 22546497, PMID 22762314, PMID 22766223, PMID 22796534, PMID 23731760, PMID 24319166, PMID 24319167, PMID 24345753, PMID 24370862, PMID 24385534, PMID 24415649, PMID 24842530, PMID 25185712, PMID 25345697, PMID 25459642, PMID 25797174, PMID 26354756, PMID 26359881, PMID 26589752, PMID 26685770, PMID 27192671, PMID 27658437, PMID 28179273, PMID 28504860, PMID 2889870, PMID 29419434, PMID 29760162, PMID 31183813
P30CA015704 (3), U10HL069294 (2), U24CA076518 (2), 2004 (1), 2014 (1), 2015 (1), 2016 (1), P01HL036444 (1), P01HL122173 (1), P30CA033572 (1), P30CA036727 (1), R01CA132197 (1), R01CA254955 (1), R01HL081499 (1), U01HL069294 (1)