| Not Yet Recruiting | Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic NCT07297550 | Institute of Hematology & Blood Diseases Hospital, China | Phase 1 / Phase 2 |
| Recruiting | CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia NCT06622694 | Institute of Hematology & Blood Diseases Hospital, China | Phase 1 |
| Recruiting | Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic An NCT06517641 | Medical College of Wisconsin | Phase 2 |
| Recruiting | Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Ane NCT06695741 | Institute of Hematology & Blood Diseases Hospital, China | Phase 2 |
| Recruiting | MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophospham NCT06412497 | Masonic Cancer Center, University of Minnesota | Phase 2 |
| Recruiting | Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anem NCT06378060 | Hematology department of the 920th hospital | Phase 2 |
| Unknown | Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are In NCT06279494 | Peking University People's Hospital | Phase 1 / Phase 2 |
| Recruiting | JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure NCT05998408 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 1 / Phase 2 |
| Active Not Recruiting | Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion NCT05236764 | Baylor College of Medicine | N/A |
| Unknown | The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA NCT06069180 | Peking University People's Hospital | Phase 4 |
| Unknown | Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired SAA Receiving sUCBT NCT06039436 | Anhui Provincial Hospital | — |
| Enrolling By Invitation | Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of NCT07299123 | Chinese PLA General Hospital | Phase 1 / Phase 2 |
| Active Not Recruiting | Cyclophosphamide Added to Standard Immunosuppressive Therapy With Herombopag as Front-line Therapy in Patients NCT05975996 | Institute of Hematology & Blood Diseases Hospital, China | Phase 2 |
| Recruiting | Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness NCT06254560 | Institute of Hematology & Blood Diseases Hospital, China | Phase 2 |
| Active Not Recruiting | A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Ane NCT05600426 | Boston Children's Hospital | Phase 3 |
| Unknown | Avatrombopag Combined With IST as First-line Treatment for SAA NCT05720234 | Institute of Hematology & Blood Diseases Hospital, China | Phase 2 |
| Unknown | A Study of PEG-rhG-CSF and rhG-CSF Used for Aplastic Anemia Granulocyte Deficiency NCT05531279 | Institute of Hematology & Blood Diseases Hospital, China | N/A |
| Unknown | Efficacy and Safety of CSA and Avatrombopag for the Treatment of SAA in the Elderly NCT05433922 | Institute of Hematology & Blood Diseases Hospital, China | N/A |
| Not Yet Recruiting | Up-front Matched Unrelated Donor Transplantation in Pediatric Patients With Idiopathic Aplastic Anemia NCT05419843 | Assistance Publique - Hôpitaux de Paris | Phase 2 |
| Recruiting | Natural History of Acquired and Inherited Bone Marrow Failure Syndromes NCT05012111 | National Heart, Lung, and Blood Institute (NHLBI) | — |
| Enrolling By Invitation | RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial NCT05049668 | European Society for Blood and Marrow Transplantation | — |
| Recruiting | AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depl NCT04099966 | Mitchell Cairo | Phase 2 |
| Recruiting | TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in NCT04356469 | Johns Hopkins All Children's Hospital | Phase 2 |
| Completed | Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy NCT04436380 | National Heart, Lung, and Blood Institute (NHLBI) | — |
| Completed | Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chro NCT04436367 | National Heart, Lung, and Blood Institute (NHLBI) | — |
| Recruiting | Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia NCT04304820 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Unknown | Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation NCT03836690 | University College, London | Phase 1 |
| Completed | Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia NCT03825744 | Jiangsu HengRui Medicine Co., Ltd. | Phase 3 |
| Unknown | Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin Regimens in Severe Apla NCT03295058 | Assiut University | Phase 2 / Phase 3 |
| Recruiting | Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders NCT03579875 | Masonic Cancer Center, University of Minnesota | Phase 2 |
| Completed | A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient NCT03557099 | Jiangsu HengRui Medicine Co., Ltd. | Phase 1 / Phase 2 |
| Terminated | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Pa NCT03333486 | Roswell Park Cancer Institute | Phase 2 |
| Active Not Recruiting | Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus H NCT03192397 | Roswell Park Cancer Institute | Phase 1 / Phase 2 |
| Unknown | Haploidentical Transplantation in Severe Aplastic Anemia NCT03246178 | Wu Xiaoxiong | N/A |
| Completed | Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502) NCT02918292 | Medical College of Wisconsin | Phase 2 |
| Recruiting | Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM NCT03173937 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 1 / Phase 2 |
| Completed | Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia NCT02998645 | Novartis Pharmaceuticals | Phase 2 |
| Active Not Recruiting | Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppres NCT02979873 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Recruiting | Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic NCT02828592 | Northside Hospital, Inc. | Phase 2 |
| Completed | NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia NCT02833805 | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Phase 2 |
| Completed | Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia NCT02845596 | Michael Pulsipher | N/A |
| Completed | The Efficacy of Immunosuppressive Therapy Combined With Cord Blood Transfusion in Treatment of Severe Aplastic NCT02745717 | Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | Phase 4 |
| Completed | hATG+CsA vs hATG+CsA+Eltrombopag for SAA NCT02099747 | European Society for Blood and Marrow Transplantation | Phase 3 |
| Unknown | Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia NCT02407470 | Navy General Hospital, Beijing | Phase 1 / Phase 2 |
| Completed | Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory NCT02224872 | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Phase 2 |
| Completed | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases NCT01917708 | Emory University | Phase 1 |
| Completed | Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis NCT02065154 | University of Alabama at Birmingham | Phase 2 |
| Completed | Human Placental-Derived Stem Cell Transplantation NCT01586455 | New York Medical College | Phase 1 |
| Active Not Recruiting | CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation NCT01966367 | Diane George | Phase 1 / Phase 2 |
| Unknown | Mesenchymal Stem Cells Co-transplantation in Alternative Donor Transplantation of Severe Aplastic Anemia. NCT02247973 | Guangzhou General Hospital of Guangzhou Military Command | Phase 2 |
| Completed | Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia NCT01703169 | University of Utah | Phase 2 |
| Active Not Recruiting | Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia NCT01623167 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 1 / Phase 2 |
| Recruiting | A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) NCT01351545 | Center for International Blood and Marrow Transplant Research | — |
| Unknown | Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation NCT01472055 | Seoul National University Hospital | Phase 2 |
| Terminated | Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia NCT01383434 | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Phase 2 |
| Recruiting | Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Sy NCT01174108 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Unknown | Different Doses of Anti-thymocyte Globin to Treat Child Severe Aplastic Anemia NCT01997372 | Chinese Academy of Medical Sciences | Phase 4 |
| Terminated | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation NCT01319851 | Emory University | N/A |
| Completed | Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia NCT01193283 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 1 / Phase 2 |
| Completed | A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia NCT01187017 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 1 / Phase 2 |
| Withdrawn | Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplasti NCT01129323 | City of Hope Medical Center | N/A |
| Completed | Unrelated Umbilical Cord Blood (UBC)Transplantation NCT01768845 | West Virginia University | N/A |
| Terminated | Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant a NCT00358657 | Fred Hutchinson Cancer Center | Phase 2 |
| Completed | Unrelated Donor Stem Cell Transplantation NCT01364363 | Scripps Health | N/A |
| Completed | Improving Immunosuppressive Treatment for Patients With Severe Aplastic Anemia NCT00061360 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorde NCT00516152 | University of California, San Francisco | Phase 2 |
| Terminated | Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia NCT00590460 | Baylor College of Medicine | Phase 1 / Phase 2 |
| Completed | Combination Therapy of Severe Aplastic Anemia NCT00001964 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syn NCT00004143 | David Rizzieri, MD | Phase 2 |
| Terminated | New York Blood Center National Cord Blood Program NCT00212407 | New York Blood Center | EARLY_Phase 1 |