| Recruiting | Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS) NCT07186179 | Northwell Health | — |
| Completed | Baby Detect : Genomic Newborn Screening NCT05687474 | Centre Hospitalier Universitaire de Liege | — |
| Completed | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marro NCT04965597 | Fred Hutchinson Cancer Center | Phase 2 |
| Recruiting | AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depl NCT04099966 | Mitchell Cairo | Phase 2 |
| Terminated | The Use of Trifluoperazine in Transfusion Dependent DBA NCT03966053 | Adrianna Vlachos, MD | Phase 1 / Phase 2 |
| Recruiting | T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias NCT03653338 | Paul Szabolcs | Phase 1 / Phase 2 |
| Completed | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation NCT03513328 | University of Florida | Phase 1 / Phase 2 |
| Recruiting | Familial Investigations of Childhood Cancer Predisposition NCT03050268 | St. Jude Children's Research Hospital | — |
| Terminated | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study NCT03733249 | Bellicum Pharmaceuticals | Phase 1 / Phase 2 |
| Recruiting | Investigation of the Genetics of Hematologic Diseases NCT02720679 | St. Jude Children's Research Hospital | — |
| Terminated | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies NCT02179359 | Masonic Cancer Center, University of Minnesota | N/A |
| Unknown | L-leucine in Diamond Blackfan Anemia Patients NCT02386267 | Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache | Phase 2 |
| Terminated | Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant NCT02065869 | Bellicum Pharmaceuticals | Phase 1 / Phase 2 |
| Completed | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases NCT01917708 | Emory University | Phase 1 |
| Completed | Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Black NCT01362595 | Northwell Health | Phase 1 / 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 |
| Completed | Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders NCT01758042 | Massachusetts General Hospital | N/A |
| Completed | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem NCT01529827 | Roswell Park Cancer Institute | Phase 2 |
| Terminated | Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia NCT01464164 | Northwell Health | Phase 1 / Phase 2 |
| Withdrawn | Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies NCT01419704 | Talaris Therapeutics Inc. | Phase 1 / Phase 2 |
| Terminated | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation NCT01319851 | Emory University | N/A |
| Completed | Multi-Center Study of Iron Overload: Survey Study (MCSIO) NCT01913548 | UCSF Benioff Children's Hospital Oakland | — |
| Completed | Multi-Center Study of Iron Overload: Pilot Study NCT01114776 | UCSF Benioff Children's Hospital Oakland | — |
| Completed | Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity C NCT00957931 | Stanford University | Phase 2 |
| Completed | Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Ind NCT00673608 | Novartis | Phase 4 |
| Terminated | Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells NCT02512679 | Children's Hospital Los Angeles | 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 | Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overl NCT00235391 | Novartis Pharmaceuticals | Phase 3 |
| Completed | Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With NCT00301834 | University of California, San Francisco | Phase 2 |
| Completed | Stem Cell Transplant for Hemoglobinopathy NCT00176852 | Masonic Cancer Center, University of Minnesota | Phase 2 / Phase 3 |
| Recruiting | Cancer in Inherited Bone Marrow Failure Syndromes NCT00027274 | National Cancer Institute (NCI) | — |
| Completed | Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia NCT00011505 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young P NCT00305708 | University of California, San Francisco | Phase 1 / Phase 2 |
| Completed | Stem Cell Transplant for Bone Marrow Failure Syndromes NCT00176878 | Masonic Cancer Center, University of Minnesota | Phase 2 / Phase 3 |
| Terminated | A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure NCT00001962 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Terminated | Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer NCT00290628 | Masonic Cancer Center, University of Minnesota | N/A |