13 completed, 12 recruiting, 7 unknown, 5 active not recruiting. 44 Phase 2, 13 Phase 1, 1 Phase 3.
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 |
| 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 |
| Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy - Severe Aplastic Anemia, Cyclophosphamide Reduced-dose | Phase 2 | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 75 | 2024-11-01 | 2024-11-19 |
| MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure. Rituximab, Rabbit ATG, Cyclophosphamide - Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia | Phase 2 | Recruiting | Masonic Cancer Center, University of Minnesota | 60 | 2024-06-05 | 2026-06-03 |
| Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia Modified transplantation system - Severe Aplastic Anemia | Phase 2 | Recruiting | Hematology department of the 920th hospital | 30 | 2024-03-01 | 2024-04-22 |
| 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 |
| Cyclophosphamide Added to Standard Immunosuppressive Therapy With Herombopag as Front-line Therapy in Patients With Severe Aplastic Anemia Cyclophosphamide added to standard immunosuppressive therapy with herombopag - Severe Aplastic Anemia | Phase 2 | Unknown | Institute of Hematology & Blood Diseases Hospital, China | 43 | 2023-07-10 | 2024-09-25 |
| Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness Rituximab - Severe Aplastic Anemia, Platelet Transfusion Refractoriness | Phase 2 | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 20 | 2023-02-23 | 2024-02-12 |
| Avatrombopag Combined With IST as First-line Treatment for SAA avatrombopag - Severe Aplastic Anemia | Phase 2 | Unknown | Institute of Hematology & Blood Diseases Hospital, China | 53 | 2022-11-10 | 2023-02-09 |
| 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 |
| Up-front Matched Unrelated Donor Transplantation in Pediatric Patients With Idiopathic Aplastic Anemia HSCT Arm group - Idiopathic Aplastic Anemia, Severe Aplastic Anemia | Phase 2 | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 25 | 2022-06-01 | 2022-06-15 |
| AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion alpha beta depletion - Acute Leukemia, Severe Aplastic Anemia | Phase 2 | Recruiting | Mitchell Cairo | 20 | 2021-04-01 | 2026-10-23 |
| TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children Haploidentical Hematopoietic Cell Transplantation - Hemoglobinopathy (Disorder), Severe Aplastic Anemia | Phase 2 | Recruiting | Johns Hopkins All Children's Hospital | 17 | 2020-07-22 | 2026-07-01 |
| Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA) Eltrombopag, Cyclosporine, Horse-Anti-thymocyte-Globulin - Severe Aplastic Anemia | Phase 2 | Recruiting | National Heart, Lung, and Blood Institute (NHLBI) | 80 | 2020-05-07 | 2026-03-09 |
| Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin Regimens in Severe Aplastic Anemia Patients Non ATG Conditioning regimen, GVHD Prophylaxis, Allogenic Stem Cell Transplantation - Severe Aplastic Anemia | Phase 2 / Phase 3 | Unknown | Assiut University | 50 | 2019-01-01 | 2018-11-27 |
| Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU) - Fanconi Anemia, Severe Aplastic Anemia | Phase 2 | Recruiting | Masonic Cancer Center, University of Minnesota | 48 | 2018-11-13 | 2026-01-30 |
| 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 |
| Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis - Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission | Phase 2 | Terminated | Roswell Park Cancer Institute | 31 | 2017-12-07 | 2025-07-04 |
| 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 |
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Anemia, Aplastic (44) · Bone Marrow Failure Disorders (7) · Myelodysplastic Syndromes (7) · Lymphoma, Non-Hodgkin (5) · Anemia, Sickle Cell (4) · Hemoglobinuria, Paroxysmal (4) · Leukemia, Myelogenous, Chronic, BCR-ABL Positive (4) · Anemia, Diamond-Blackfan (3) · Autoimmune Diseases (3) · Hodgkin Disease (3) · Leukemia, Myeloid, Acute (3) · Multiple Myeloma (3) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (3) · Pancytopenia (3) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (3)
Anemia (44) · Bone Marrow Diseases (44) · Hematologic Diseases (44) · Hemic and Lymphatic Diseases (44) · Bone Marrow Failure Disorders (37) · Immune System Diseases (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Genetic Diseases, Inborn (7) · Anemia, Hemolytic (6) · Immunoproliferative Disorders (6) · Lymphatic Diseases (6) · Anemia, Hypoplastic, Congenital (5) · Congenital Bone Marrow Failure Syndromes (5) · Cytopenia (5) · Lymphoproliferative Disorders (5)
Cyclophosphamide (12) · fludarabine (9) · Whole-Body Irradiation (8) · Mycophenolic Acid (6) · Cyclosporine (5) · Tacrolimus (5) · fludarabine phosphate (5) · thymoglobulin (5) · Sirolimus (4) · eltrombopag (4) · Alemtuzumab (3) · Antilymphocyte Serum (3) · Rituximab (3) · Bone Marrow Transplantation (2) · Busulfan (2)
National Heart, Lung, and Blood Institute (NHLBI) (10) · Institute of Hematology & Blood Diseases Hospital, China (5) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (3) · Masonic Cancer Center, University of Minnesota (2) · Medical College of Wisconsin (2) · Roswell Park Cancer Institute (2) · Assistance Publique - Hôpitaux de Paris (1) · Assiut University (1) · Baylor College of Medicine (1) · Chinese PLA General Hospital (1) · David Rizzieri, MD (1) · Diane George (1) · Fred Hutchinson Cancer Center (1) · Guangzhou General Hospital of Guangzhou Military Command (1) · Hematology department of the 920th hospital (1)
United States (29) · China (9) · South Korea (2) · Brazil (1) · Hong Kong (1) · India (1) · Italy (1) · Mexico (1) · Spain (1) · Thailand (1) · Turkey (Türkiye) (1)
Neal S Young, M.D. (3) · Bhavisha A Patel, M.D. (2) · Danielle M Townsley, M.D. (2) · Richard W Childs, M.D. (2) · Amy DeZern, MD (1) · Amy Dezern, MD (1) · Amy E DeZern, MD (1) · Bhavisha Patel, M.D. (1) · David A. Rizzieri, MD (1) · Deepak Chellapandian, MD (1) · Diane George, MD (1) · George M Rodgers, M.D. (1) · Huang Jinbo, MD. (1) · Hyoung Jin Kang, MD., PhD (1) · Kanwaldeep Mallhi (1)
PMID 34724566, PMID 32343796, PMID 19181786, PMID 22517900, PMID 22796534, PMID 24319166, PMID 24319167, PMID 24842530, PMID 25185712, PMID 25797174, PMID 26354756, PMID 26359881, PMID 2889870, PMID 29419434, PMID 29674506, PMID 29958797, PMID 33410523, PMID 33910334, PMID 34288026, PMID 34525188, PMID 35371427, PMID 35907408, PMID 37084383, PMID 38335978
2016 (1), P01HL122173 (1), P30CA015704 (1), U10HL069294 (1), U24CA076518 (1)