5 completed, 4 active not recruiting, 3 unknown, 1 enrolling by invitation. 17 Phase 1, 13 Phase 2.
Follow Severe Aplastic Anemia: new trials by email Literature Review of Severe Aplastic Anemia trials Research Report on Severe 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 |
|---|---|---|---|---|---|---|
| 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 |
| CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia RD13-02 - Severe Aplastic Anemia, Relapse | Phase 1 | Suspended | Institute of Hematology & Blood Diseases Hospital, China | 15 | 2025-06-19 | 2026-11-04 |
| 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 |
| 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 |
| Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation CD62L- Tem - Lymphoma, Leukemia | Phase 1 | Unknown | University College, London | 18 | 2019-10-21 | 2019-10-25 |
| A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient Hetrombopag Olamine - Severe Aplastic Anemia | Phase 1 / Phase 2 | Completed | Jiangsu HengRui Medicine Co., Ltd. | 55 | 2018-06-20 | 2022-07-29 |
| Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide, Fludarabine Phosphate - Acute Myeloid Leukemia in Remission, Adult Acute Lymphoblastic Leukemia in Complete Remission | Phase 1 / Phase 2 | Active not recruiting | Roswell Park Cancer Institute | 35 | 2017-08-09 | 2026-04-14 |
| Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome Omidubicel (former CordIn), Miltenyi CliniMACS(R) CD34 Reagent System - Severe Aplastic Anemia, Hypo-Plastic MDS | Phase 1 / Phase 2 | Recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 37 | 2017-06-13 | 2026-10-01 |
| Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia Rabbit antithymoglobulin (ATG), Adipose derived mesenchymal stem cells ( AD-MSCs), AD-MSC transdifferentiated HSCs (AD-HSCs) - Severe Aplastic Anemia | Phase 1 / Phase 2 | Unknown | Navy General Hospital, Beijing | 90 | 2015-01-01 | 2015-04-03 |
| Bone Marrow Transplant With Abatacept for Non-Malignant Diseases Abatacept - Hurler Syndrome, Fanconi Anemia | Phase 1 | Completed | Emory University | 10 | 2014-01-01 | 2019-12-26 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation CD34 Stem Cell Selection Therapy - Bone Marrow Failure Syndrome, Severe Aplastic Anemia | Phase 1 / Phase 2 | Active not recruiting | Diane George | 37 | 2013-03-01 | 2025-08-14 |
| Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia Cohort 1: hATG, CsA, EPAG Day 14 to Month 6, Cohort 2: hATG, CsA, EPAG Day 14 to Month 3, Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6 - Severe Aplastic Anemia | Phase 1 / Phase 2 | Active not recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 207 | 2012-07-02 | 2026-04-13 |
| Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia Cyclophosphamide, Cyclosporine - Aplastic Anemia, Neutropenia | Phase 1 / Phase 2 | Completed | National Heart, Lung, and Blood Institute (NHLBI) | 22 | 2010-08-01 | 2017-05-22 |
| A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia Cyclophosphamide, Fludarabine - Aplastic Anemia, Neutropenia | Phase 1 / Phase 2 | Completed | National Heart, Lung, and Blood Institute (NHLBI) | 1 | 2010-08-01 | 2021-07-02 |
| Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia CAMPATH-1H, Anti-CD45, Fludarabine - Fanconi Anemia, Severe Aplastic Anemia | Phase 1 / Phase 2 | Terminated | Baylor College of Medicine | 5 | 2001-07-01 | 2018-05-24 |
Anemia, Aplastic (17) · Myelodysplastic Syndromes (5) · Anemia, Diamond-Blackfan (3) · Graft vs Host Disease (3) · Pancytopenia (3) · Anemia, Sickle Cell (2) · Autoimmune Diseases (2) · Fanconi Anemia (2) · Lymphohistiocytosis, Hemophagocytic (2) · Mucopolysaccharidosis I (2) · Neutropenia (2) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (2) · Primary Immunodeficiency Diseases (2) · Shwachman-Diamond Syndrome (2) · Acquired Immunodeficiency Syndrome (1)
Anemia (17) · Bone Marrow Diseases (17) · Hematologic Diseases (17) · Hemic and Lymphatic Diseases (17) · Bone Marrow Failure Disorders (16) · Immune System Diseases (8) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Lymphatic Diseases (5) · Anemia, Hypoplastic, Congenital (4) · Congenital Bone Marrow Failure Syndromes (4) · Cytopenia (4) · Immunologic Deficiency Syndromes (4) · Immunoproliferative Disorders (4) · Lymphoproliferative Disorders (4)
Cyclophosphamide (3) · Abatacept (2) · Cyclosporine (2) · Sirolimus (2) · fludarabine (2) · Alemtuzumab (1) · Cyclosporins (1) · Influenza Vaccines (1) · Melphalan (1) · Mycophenolic Acid (1) · Whole-Body Irradiation (1) · eltrombopag (1) · fludarabine phosphate (1) · ruxolitinib (1)
National Heart, Lung, and Blood Institute (NHLBI) (5) · Institute of Hematology & Blood Diseases Hospital, China (2) · Baylor College of Medicine (1) · Chinese PLA General Hospital (1) · Diane George (1) · Emory University (1) · Jiangsu HengRui Medicine Co., Ltd. (1) · Navy General Hospital, Beijing (1) · New York Medical College (1) · Peking University People's Hospital (1) · Roswell Park Cancer Institute (1) · University College, London (1)
United States (10) · China (4) · United Kingdom (1)
Danielle M Townsley, M.D. (2) · Bhavisha Patel, M.D. (1) · Diane George, MD (1) · John T Horan, MD (1) · Malcolm Brenner, M.B., Ph.D., (1) · Maureen Ross (1) · Mitchell S Cairo, MD (1) · Neal S Young, M.D. (1) · Richard W Childs, M.D. (1) · Xiao-Jun Huang (1)
PMID 34724566, PMID 25185712, PMID 26354756, PMID 29419434, PMID 29674506, PMID 29958797, PMID 33410523, PMID 33910334, PMID 34525188, PMID 35371427