| Unknown | Efficacy and Safety of Avatrombopag vs. Avatrombopag Combined With rhTPO in the Treatment of SAA NCT06254287 | Institute of Hematology & Blood Diseases Hospital, China | Phase 2 |
| Completed | Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Medical Claims Database Study NCT06991894 | Novartis Pharmaceuticals | — |
| Terminated | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study NCT03733249 | Bellicum Pharmaceuticals | Phase 1 / Phase 2 |
| Unknown | Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia NCT02857530 | Tianjin Medical University General Hospital | Phase 2 |
| Completed | Efficacy and Safety of Eltrombopag + CSA in Patients With Moderate Aplastic Anemia (EMAA) NCT02773225 | B. Höchsmann | Phase 2 / Phase 3 |
| Terminated | Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant NCT02065869 | Bellicum Pharmaceuticals | Phase 1 / Phase 2 |
| Active Not Recruiting | Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant NCT03301168 | Bellicum Pharmaceuticals | Phase 1 / Phase 2 |
| Completed | Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States NCT00997386 | University of Arizona | Phase 2 |
| Completed | Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After Nonmyeloa NCT00975975 | Indiana University School of Medicine | Phase 2 |
| Completed | Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Trea NCT00944749 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients NCT00922883 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors NCT00618969 | University of Arizona | Phase 2 |
| Completed | Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia NCT00578266 | Mayo Clinic | Phase 1 |
| Terminated | Study of Parathyroid Hormone Following Sequential Cord Blood Transplantation From an Unrelated Donor NCT00393380 | The Emmes Company, LLC | Phase 2 |
| Unknown | Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia NCT00319878 | Office of Rare Diseases (ORD) | Phase 1 / Phase 2 |
| Unknown | Identifying Characteristics of Bone Marrow Failure Syndromes NCT00315419 | Office of Rare Diseases (ORD) | — |
| Unknown | Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blo NCT00737685 | The Korean Society of Pediatric Hematology Oncology | Phase 2 |
| Completed | Fludarabine-based Conditioning for Severe Aplastic Anemia (BMT CTN 0301) NCT00326417 | Medical College of Wisconsin | Phase 1 / Phase 2 |
| Completed | Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory NCT00244010 | St. Jude Children's Research Hospital | N/A |
| Completed | Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells NCT00692926 | Joanne Kurtzberg, MD | Phase 1 |
| Completed | Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia NCT00229619 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic NCT00061763 | Novartis Pharmaceuticals | Phase 2 |
| Unknown | Safety and Efficacy Study of Ex Vivo Immunotherapy for Treatment of Aplastic Anemia NCT00399971 | Shenzhen Zhongxing Yangfan Biotech Co. Ltd. | Phase 1 / Phase 2 |
| Completed | T-Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Hematologic Malignancies NCT00683046 | University of Chicago | Phase 2 |
| Completed | Cord Blood Stem Cell Transplantation Study (COBLT) NCT00000603 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Conditioning Regimens for Patients With Severe Aplastic Anemia Transplanted With Marrow From an Unrelated Dono NCT00144729 | Fred Hutchinson Cancer Center | Phase 1 / Phase 2 |
| Completed | Etiology of Blood Dyscrasias: Analysis of the International Agranulocytosis and Aplastic Anemia Study Data NCT00005307 | National Heart, Lung, and Blood Institute (NHLBI) | — |
| Completed | Aplastic Anemia Epidemiology: Incidence and Case-control NCT00005682 | National Heart, Lung, and Blood Institute (NHLBI) | — |
| Completed | Drug Etiology of Aplastic Anemia and Related Dyscrasias NCT00005302 | National Heart, Lung, and Blood Institute (NHLBI) | — |
| Completed | Multi-Center Trial of Anti-Thymocyte Globulin in Treatment of Aplastic Anemia and Other Hematologic Disorders NCT00000597 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 3 |