69 completed, 36 unknown, 17 recruiting, 11 terminated. 74 Phase 2, 25 Phase 1, 19 Not applicable, 14 Phase 4, 13 Phase 3.
Follow Aplastic Anemia: new trials by email Literature Review of Aplastic Anemia trials Research Report on Aplastic Anemia
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 |
| CK0801 for Treatment of Aplastic Anemia CK0801 - Aplastic Anemia | Phase 2 | Not yet recruiting | Cellenkos, Inc. | 12 | 2026-12-03 | 2026-10-20 |
| Prophylactic NAC to Improve Platelet Engraftment After Haploidentical Transplantation in Severe Aplastic Anemia Patients N-acetylcysteine - Aplastic Anemia | Phase 3 | Not yet recruiting | Peking University People's Hospital | 142 | 2026-09-30 | 2026-09-16 |
| 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N01 in the Treatment of Refractory Chemoradiotherapy-induced AA Romiplostim N01 - Aplastic Anemia | Phase 2 | Withdrawn | Peking Union Medical College Hospital | 2026-06-01 | 2026-06-04 | |
| 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 |
| 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 |
| A Prediction Model in Pregnant Women With Aplastic Anemia A combined retrospective and prospective observational follow-up - Aplastic Anemia, Pregnancy | Recruiting | Peking University People's Hospital | 300 | 2025-11-27 | 2025-12-01 | |
| Brain Cognitive Network Abnormalities in Anemia Patients Using fNIRS Aplastic Anemia, Myelodysplastic Syndromes | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 323 | 2025-10-11 | 2026-09-18 | |
| 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 |
| 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 | |
| Reproductive and Mental Health of Patients With Aplastic Anemia Sex hormone monitoring - Aplastic Anemia, Reproductive Health | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 600 | 2025-03-03 | 2025-08-05 | |
| 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 |
| Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years Allogeneic hematopoietic stem cell transplantation Stem cell source only Bone Marrow - Aplastic Anemia | Phase 2 | Recruiting | Assistance Publique - Hôpitaux de Paris | 52 | 2025-01-24 | 2026-04-28 |
| Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy Let's Get REAL - Hematologic Malignancy, Solid Tumor | Not applicable | Recruiting | Washington University School of Medicine | 60 | 2024-11-26 | 2026-06-04 |
| Effects of Levetiracetam Monotherapy on Various Hematological Parameters in Adult Patients Suffering of Epilepsy Neutropenia, Thrombocytopenia | Unknown | Assiut University | 45 | 2024-11-01 | 2024-07-09 | |
| Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia standard IST combined with Romiplostim N01 - Aplastic Anemia, TPO Receptor Agonists | Phase 2 | Unknown | Institute of Hematology & Blood Diseases Hospital, China | 48 | 2024-10-24 | 2024-12-04 |
| CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia CD7 CAR-T cells injection, Allogeneic hematopoietic stem cell transplantation - Aplastic Anemia | Early Phase 1 | Recruiting | Zhejiang University | 30 | 2024-10-20 | 2024-10-09 |
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Anemia, Aplastic (155) · Myelodysplastic Syndromes (35) · Multiple Myeloma (26) · Leukemia, Myeloid, Acute (24) · Hodgkin Disease (21) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (20) · Leukemia, Lymphocytic, Chronic, B-Cell (18) · Lymphoma, Non-Hodgkin (18) · Myeloproliferative Disorders (16) · Bone Marrow Failure Disorders (15) · Leukemia (15) · Leukemia, Myelogenous, Chronic, BCR-ABL Positive (14) · Lymphoma (13) · Primary Myelofibrosis (13) · Leukemia, Myelomonocytic, Chronic (10)
Hemic and Lymphatic Diseases (155) · Bone Marrow Diseases (154) · Anemia (151) · Hematologic Diseases (149) · Bone Marrow Failure Disorders (140) · Immune System Diseases (42) · Neoplasms (40) · Neoplasms by Histologic Type (40) · Pathological Conditions, Signs and Symptoms (36) · Immunoproliferative Disorders (35) · Lymphatic Diseases (34) · Lymphoproliferative Disorders (34) · Pathologic Processes (34) · Disease Attributes (30) · Blood Protein Disorders (27)
Cyclophosphamide (35) · Cyclosporine (26) · fludarabine (23) · Antilymphocyte Serum (21) · thymoglobulin (20) · Whole-Body Irradiation (17) · fludarabine phosphate (17) · Mycophenolic Acid (12) · Tacrolimus (12) · Peripheral Blood Stem Cell Transplantation (9) · Busulfan (8) · Cyclosporins (8) · Granulocyte Colony-Stimulating Factor (8) · romiplostim (8) · Alemtuzumab (7)
Peking Union Medical College Hospital (15) · Institute of Hematology & Blood Diseases Hospital, China (9) · National Heart, Lung, and Blood Institute (NHLBI) (9) · M.D. Anderson Cancer Center (6) · Novartis Pharmaceuticals (6) · Roswell Park Cancer Institute (5) · Kyowa Kirin Co., Ltd. (4) · Peking University People's Hospital (4) · Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University (4) · Fred Hutchinson Cancer Center (3) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (3) · Anjali Sharathkumar (2) · Beth Israel Deaconess Medical Center (2) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (2) · King's College Hospital NHS Trust (2)
United States (71) · China (37) · Japan (8) · South Korea (7) · United Kingdom (4) · Brazil (3) · Germany (3) · Italy (3) · Russia (3) · France (2) · Switzerland (2) · Australia (1) · Austria (1) · Canada (1) · Egypt (1)
Bing Bing, PhD (3) · Danielle M Townsley, M.D. (3) · Usama Gergis, MD (3) · Bing Han, PhD (2) · David E Avigan, MD (2) · David F McDermott, MD (2) · George Chen (2) · Jun Shi, PhD (2) · Neal Flomenberg, MD (2) · Akshay Sharma, MBBS (1) · Alan H DeCherney, M.D. (1) · Alexander Ngwube, MD (1)
PMID 40593138, PMID 1090310, PMID 12622583, PMID 18379007, PMID 20383216, PMID 20685256, PMID 21606164, PMID 21663651, PMID 22055111, PMID 22067384, PMID 22526363, PMID 22544699, PMID 23731760, PMID 24838091, PMID 25185712, PMID 25310303, PMID 25932194, PMID 26589752, PMID 26622526, PMID 27192671, PMID 28179273, PMID 30504345, PMID 31151984, PMID 31474546, PMID 31807987, PMID 31851762, PMID 32343796, PMID 33305842, PMID 33875811, PMID 34086408, PMID 34724566, PMID 35590143, PMID 35854224, PMID 35911824, PMID 36173561, PMID 36326854, PMID 36441357, PMID 36579443, PMID 36647781, PMID 37084383, PMID 37318085, PMID 37534514, PMID 37680953, PMID 37846845, PMID 37919313, PMID 37967471, PMID 38170583, PMID 38249329, PMID 38467031, PMID 38505288
2015 (1), 2019 (1), K12CA167540 (1), P01HL036444 (1), R01CA254955 (1)