38 completed, 11 unknown, 7 recruiting, 6 not yet recruiting. 74 Phase 2, 16 Phase 1, 7 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 |
|---|---|---|---|---|---|---|
| CK0801 for Treatment of Aplastic Anemia CK0801 - Aplastic Anemia | Phase 2 | Not yet recruiting | Cellenkos, Inc. | 12 | 2026-12-03 | 2026-10-20 |
| 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N01 in the Treatment of Refractory Chemoradiotherapy-induced AA Romiplostim N01 - Aplastic Anemia | Phase 2 | Withdrawn | Peking Union Medical College Hospital | 2026-06-01 | 2026-06-04 | |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 |
| 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 | |
| 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 |
| 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 |
| NAC for Hematopoietic Recovery in SAA N Acetyl L Cysteine - Aplastic Anemia | Phase 2 | Unknown | Peking University People's Hospital | 30 | 2024-07-25 | 2024-07-24 |
| A Study of Emapalumab for Pediatric Aplastic Anemia Emapalumab - Aplastic Anemia, Cytopenia | Phase 2 | Recruiting | Memorial Sloan Kettering Cancer Center | 35 | 2024-05-21 | 2025-12-17 |
| Efficacy and Safety of CsA+AVA in the Treatment of NSAA in the Elderly Ciclosporin, Avatrombopag - Aplastic Anemia | Phase 2 | Unknown | Peking Union Medical College Hospital | 30 | 2023-08-17 | 2023-12-08 |
| Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors Nicotinamide Riboside, Placebo, Exercise Intervention - Lymphoblastic Leukemia, Cancer Survivors | Phase 2 | Recruiting | Children's Hospital of Philadelphia | 80 | 2023-02-23 | 2025-12-04 |
| Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia Luspatercept, Cyclosporine - Aplastic Anemia | Phase 2 | Completed | Bing Han | 58 | 2022-12-19 | 2025-09-16 |
| GVHD Prophylaxis by Addition of CD20 Monoclonal Antibody to the Conditioning Regimen in SAA With Treatment of Allo-HSCT CD20 monoclonal antibody, ATG - Aplastic Anemia | Phase 2 | Unknown | The First Affiliated Hospital of Soochow University | 100 | 2021-12-30 | 2022-08-24 |
| Two Step Haplo With Radiation Conditioning Fludarabine, Total-Body Irradiation, Donor Lymphocyte Infusion - Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia | Phase 2 | Recruiting | Thomas Jefferson University | 63 | 2021-10-25 | 2025-10-30 |
| Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia Hetrombopag - Aplastic Anemia, Drug Effect | Phase 2 / Phase 3 | Withdrawn | Peking Union Medical College Hospital | 2021-10-01 | 2026-06-08 |
Showing 20 of 74. Sign in to see all of them.
Anemia, Aplastic (74) · Myelodysplastic Syndromes (17) · Multiple Myeloma (13) · Hodgkin Disease (12) · Leukemia, Myeloid, Acute (11) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (10) · Bone Marrow Failure Disorders (9) · Leukemia, Lymphocytic, Chronic, B-Cell (9) · Lymphoma, Non-Hodgkin (9) · Myeloproliferative Disorders (8) · Leukemia, Myelogenous, Chronic, BCR-ABL Positive (7) · Leukemia, Myelomonocytic, Chronic (7) · Lymphoma, Follicular (6) · Primary Myelofibrosis (6) · Recurrence (6)
Hemic and Lymphatic Diseases (74) · Bone Marrow Diseases (73) · Anemia (72) · Hematologic Diseases (71) · Bone Marrow Failure Disorders (65) · Pathological Conditions, Signs and Symptoms (21) · Immune System Diseases (20) · Pathologic Processes (20) · Neoplasms (18) · Neoplasms by Histologic Type (18) · Disease Attributes (17) · Immunoproliferative Disorders (17) · Lymphatic Diseases (17) · Lymphoproliferative Disorders (17) · Blood Protein Disorders (13)
Cyclophosphamide (23) · Cyclosporine (18) · fludarabine (16) · Antilymphocyte Serum (13) · Whole-Body Irradiation (13) · fludarabine phosphate (12) · Mycophenolic Acid (11) · Tacrolimus (11) · thymoglobulin (11) · Peripheral Blood Stem Cell Transplantation (8) · Methylprednisolone (6) · Alemtuzumab (5) · Busulfan (5) · Cyclosporins (5) · Filgrastim (5)
National Heart, Lung, and Blood Institute (NHLBI) (6) · Peking Union Medical College Hospital (6) · Institute of Hematology & Blood Diseases Hospital, China (4) · Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University (4) · Kyowa Kirin Co., Ltd. (3) · M.D. Anderson Cancer Center (3) · Novartis Pharmaceuticals (3) · Roswell Park Cancer Institute (3) · Beth Israel Deaconess Medical Center (2) · Fred Hutchinson Cancer Center (2) · Memorial Sloan Kettering Cancer Center (2) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (2) · The Korean Society of Pediatric Hematology Oncology (2) · University of Sao Paulo (2) · Anjali Sharathkumar (1)
United States (38) · China (14) · South Korea (6) · Japan (4) · Germany (3) · Brazil (2) · France (2) · United Kingdom (2) · Austria (1) · Hong Kong (1) · Italy (1) · Norway (1) · Portugal (1) · Russia (1) · Saudi Arabia (1)
Danielle M Townsley, M.D. (3) · Usama Gergis, MD (3) · David E Avigan, MD (2) · David F McDermott, MD (2) · Jun Shi, PhD (2) · Neal Flomenberg, MD (2) · Akshay Sharma, MBBS (1) · Amr Qudeimat, MD (1) · Amy E DeZern, MD (1) · Ana Beatriz Hortense, MD (1) · Andreas Klein, MD (1) · Andromachi Scaradavou, MD (1) · Anjali Sharathkumar, MD (1)
PMID 40593138, PMID 1090310, PMID 12622583, PMID 20383216, PMID 20685256, PMID 21606164, PMID 22055111, PMID 22067384, PMID 22544699, PMID 23731760, PMID 25185712, PMID 25310303, PMID 26589752, PMID 27192671, PMID 28179273, PMID 31474546, PMID 31851762, PMID 32343796, PMID 34086408, PMID 34724566, PMID 35854224, PMID 36579443, PMID 37084383, PMID 37318085, PMID 37919313, PMID 38467031, PMID 40315366, PMID 42057135
2015 (1), P01HL036444 (1), R01CA254955 (1)