| Not Yet Recruiting | The HOPE Biobank Resource (BMT CTN 2402 HOPE) NCT07227155 | Medical College of Wisconsin | — |
| Recruiting | Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HS NCT06839456 | Children's Hospital of Philadelphia | Phase 1 / Phase 2 |
| Recruiting | Allo HSCT for High Risk Hemoglobinopathies NCT06872333 | Masonic Cancer Center, University of Minnesota | Phase 2 |
| Enrolling By Invitation | A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Wh NCT06363760 | Editas Medicine, Inc. | — |
| Active Not Recruiting | Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion NCT05236764 | Baylor College of Medicine | N/A |
| Recruiting | Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease NCT06107400 | The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army | EARLY_Phase 1 |
| Completed | Prevalence of Hemoglobinopathies Associated With Significant Hb Variants in the Chattogram Region of Banglades NCT07207551 | Bangladesh Bioscience Research Group | — |
| Recruiting | Study of the Role of Genetic Modifiers in Hemoglobinopathies NCT05799118 | Cyprus Institute of Neurology and Genetics | — |
| Recruiting | Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thal NCT05477563 | Vertex Pharmaceuticals Incorporated | Phase 3 |
| Recruiting | Discarded Bone Marrow for Hematology Research NCT04671212 | St. Jude Children's Research Hospital | — |
| Active Not Recruiting | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia NCT05356195 | Vertex Pharmaceuticals Incorporated | Phase 3 |
| Active Not Recruiting | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) NCT05329649 | Vertex Pharmaceuticals Incorporated | Phase 3 |
| Active Not Recruiting | EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent B NCT05444894 | Editas Medicine, Inc. | Phase 1 / Phase 2 |
| Active Not Recruiting | Long Term Beta Thalassemia Treatment: Findings From The Extension Period NCT06490601 | National Institute of Blood and Marrow Transplant (NIBMT), Pakistan | Phase 2 |
| Active Not Recruiting | A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) NCT04853576 | Editas Medicine, Inc. | Phase 1 / Phase 2 |
| Unknown | Transcranial Doppler in Children With Hemoglobinopathies NCT04798157 | Sohag University | N/A |
| Enrolling By Invitation | A Long-term Follow-up Study in Participants Who Received CTX001 NCT04208529 | Vertex Pharmaceuticals Incorporated | Phase 3 |
| Completed | Prevalence and Hematological Characteristics of Hemoglobinopathies NCT07180836 | Bacha Khan Medical College | — |
| Completed | Prevalence of Abnormal Haemoglobin Variants in Metropolitan Chattogram, Bangladesh NCT07305181 | Bangladesh Bioscience Research Group | — |
| Recruiting | Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders NCT04644016 | Memorial Sloan Kettering Cancer Center | Phase 2 |
| Completed | Pediatrics HOT COVID-19 Database in NY Tristate NCT04445402 | Columbia University | — |
| Completed | ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19 NCT06831799 | Hospital Universitari Vall d'Hebron Research Institute | — |
| Completed | Screening for Hemoglobinopathies in Pregnant Women NCT04029142 | University Hospital, Basel, Switzerland | — |
| Active Not Recruiting | A Blood Stem Cell Transplant for Sickle Cell Disease NCT03249831 | City of Hope Medical Center | Phase 1 |
| Completed | A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease NCT03745287 | Vertex Pharmaceuticals Incorporated | Phase 2 / Phase 3 |
| Completed | A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia NCT03655678 | Vertex Pharmaceuticals Incorporated | Phase 2 / Phase 3 |
| Terminated | In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM) NCT02986698 | University of California, San Francisco | Phase 1 |
| Withdrawn | Comparing CGM and OGTT in Relation to Iron Overload Detected by Pancreas T2* MRI in High-Risk Hematology Group NCT03141398 | Hamad Medical Corporation | — |
| Completed | Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients NCT03609840 | University of California, San Francisco | — |
| Recruiting | Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders NCT03128996 | Washington University School of Medicine | Phase 1 / Phase 2 |
| Completed | Hemoglobinopathy Nursing Program and Pediatric Nursing Students NCT05389891 | Beni-Suef University | N/A |
| Terminated | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study NCT03733249 | Bellicum Pharmaceuticals | Phase 1 / Phase 2 |
| Completed | Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT) NCT03609814 | University of California, San Francisco | — |
| Completed | Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients NCT03609827 | University of California, San Francisco | — |
| Completed | Lucky Iron Fish Home Fortification of Iron NCT02341586 | University of British Columbia | N/A |
| Completed | Hepatitis C Virus Infection in Patients With Hemoglobinopathies NCT03149289 | Società Italiana Talassemie ed Emoglobinopatie | — |
| Terminated | Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases NCT02231710 | Bellicum Pharmaceuticals | Phase 1 |
| Terminated | Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant NCT02065869 | Bellicum Pharmaceuticals | Phase 1 / Phase 2 |
| Unknown | Non-Myeloablative Conditioning and Bone Marrow Transplantation NCT01850108 | Vanderbilt-Ingram Cancer Center | N/A |
| Active Not Recruiting | CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation NCT01966367 | Diane George | Phase 1 / Phase 2 |
| Recruiting | Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC) NCT01666080 | Masonic Cancer Center, University of Minnesota | N/A |
| Completed | Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation NCT01316549 | University of California, San Francisco | — |
| Unknown | Interferon and Ribavirin Treatment in Patients With Hemoglobinopathies NCT00887081 | Azienda Ospedaliera V. Cervello | Phase 4 |
| Completed | Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Di NCT00744692 | Duke University | Phase 1 |
| Active Not Recruiting | Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders NCT01050855 | Children's Hospital of Philadelphia | 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 | Clinical and Laboratory Investigation of Humans With Informative Iron or Erythroid Phenotypes NCT00102245 | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | — |
| Completed | Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies NCT00153985 | Dana-Farber Cancer Institute | Phase 2 |
| Completed | A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regime NCT00427661 | University of Pittsburgh | N/A |
| Recruiting | Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases NCT00920972 | Washington University School of Medicine | Phase 1 / Phase 2 |
| Terminated | Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia NCT00034528 | National Institute of Allergy and Infectious Diseases (NIAID) | Phase 2 |
| Completed | Stroke Prevention in Sickle Cell Anemia (STOP 1) NCT00000592 | Augusta University | Phase 3 |
| Completed | Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG) NCT00000602 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Multicenter Study of Hydroxyurea in Patients With Sickle Cell Anemia (MSH) NCT00000586 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 3 |
| Completed | Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone NCT00000588 | Case Western Reserve University | Phase 2 |
| Completed | Penicillin Prophylaxis in Sickle Cell Disease (PROPS) NCT00000585 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 3 |
| Completed | Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis NCT00000595 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |