| NCT04356469
| TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children | | treatment | 2 | recruiting |
| NCT02349906
| Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases | | treatment | 2 | completed |
| NCT04746066
| Observational Study for Patients With Hemoglobinopathies and Rare Inherited Anemia and Covid 19 | No drug interventions | Not Available | Not Available | recruiting |
| NCT06313398
| Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling | | other | 0 | recruiting |
| NCT01200017
| Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant | No drug interventions | Not Available | Not Available | no_longer_available |
| NCT00001958
| Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia) | | treatment | 2 | completed |
| NCT00968864
| T-cell Depleted Alternative Donor Transplantation | No drug interventions | treatment | 2 | terminated |
| NCT00040417
| Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies | | treatment | 2 | terminated |
| NCT00040469
| Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies | | treatment | 2 | terminated |
| NCT05329649
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) | | treatment | 3 | recruiting |
| NCT05356195
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) | | treatment | 3 | recruiting |
| NCT04853576
| A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) | No drug interventions | treatment | 1 / 2 | active_not_recruiting |
| NCT03655678
| A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia | | treatment | 2 / 3 | active_not_recruiting |
| NCT06363760
| A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301 | No drug interventions | Not Available | Not Available | enrolling_by_invitation |
| NCT05236764
| Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion | No drug interventions | treatment | Not Available | recruiting |
| NCT04445402
| Pediatrics HOT COVID-19 Database in NY Tristate | No drug interventions | Not Available | Not Available | completed |
| NCT00427661
| A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen | No drug interventions | treatment | Not Available | completed |
| NCT00034528
| Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia | | treatment | 2 | terminated |
| NCT00153985
| Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies | | treatment | 2 | completed |
| NCT01316549
| Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation | | Not Available | Not Available | completed |
| NCT04644016
| Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders | | treatment | 2 | recruiting |
| NCT02341586
| Lucky Iron Fish Home Fortification of Iron | No drug interventions | prevention | Not Available | completed |
| NCT04798157
| Transcranial Doppler in Children With Hemoglobinopathies | No drug interventions | diagnostic | Not Available | unknown_status |
| NCT03609827
| Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | | Not Available | Not Available | completed |
| NCT05444894
| EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) | No drug interventions | treatment | 1 / 2 | recruiting |
| NCT01666080
| Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC) | | treatment | Not Available | recruiting |
| NCT04208529
| A Long-term Follow-up Study in Participants Who Received CTX001 | | other | 3 | enrolling_by_invitation |
| NCT03149289
| Hepatitis C Virus Infection in Patients With Hemoglobinopathies | No drug interventions | Not Available | Not Available | completed |
| NCT00000592
| Stroke Prevention in Sickle Cell Anemia (STOP 1) | No drug interventions | prevention | 3 | completed |
| NCT03609840
| Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | | Not Available | Not Available | completed |
| NCT01966367
| CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation | No drug interventions | treatment | 1 / 2 | active_not_recruiting |
| NCT00920972
| Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases | No drug interventions | treatment | 1 / 2 | recruiting |
| NCT02065869
| Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant | | treatment | 1 / 2 | terminated |
| NCT04671212
| Discarded Bone Marrow for Hematology Research | No drug interventions | Not Available | Not Available | recruiting |
| NCT00887081
| Interferon and Ribavirin Treatment in Patients With Hemoglobinopathies | | health_services_research | 4 | unknown_status |
| NCT00000595
| Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis | | treatment | 2 | completed |
| NCT01050855
| Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders | | treatment | 2 | active_not_recruiting |
| NCT00000602
| Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG) | | treatment | 2 | completed |
| NCT03745287
| A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease | | treatment | 2 / 3 | active_not_recruiting |
| NCT00102245
| Clinical and Laboratory Investigation of Humans With Informative Iron or Erythroid Phenotypes | No drug interventions | Not Available | Not Available | terminated |
| NCT03141398
| Comparing CGM and OGTT in Relation to Iron Overload Detected by Pancreas T2* MRI in High-Risk Hematology Group | No drug interventions | Not Available | Not Available | withdrawn |
| NCT02986698
| In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM) | No drug interventions | treatment | 1 | enrolling_by_invitation |
| NCT00000588
| Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone | No drug interventions | treatment | 2 | completed |
| NCT00000585
| Penicillin Prophylaxis in Sickle Cell Disease (PROPS) | | prevention | 3 | completed |
| NCT06107400
| Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease | No drug interventions | treatment | 0 | recruiting |
| NCT04029142
| Screening for Hemoglobinopathies in Pregnant Women | No drug interventions | Not Available | Not Available | completed |
| NCT03733249
| Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study | | treatment | 1 / 2 | terminated |
| NCT05799118
| Study of the Role of Genetic Modifiers in Hemoglobinopathies | No drug interventions | Not Available | Not Available | recruiting |
| NCT03128996
| Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders | No drug interventions | treatment | 1 / 2 | recruiting |
| NCT05477563
| Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | | treatment | 3 | recruiting |
| NCT00000586
| Multicenter Study of Hydroxyurea in Patients With Sickle Cell Anemia (MSH) | | treatment | 3 | completed |
| NCT03249831
| A Blood Stem Cell Transplant for Sickle Cell Disease | | treatment | 1 | active_not_recruiting |
| NCT05389891
| Hemoglobinopathy Nursing Program and Pediatric Nursing Students | No drug interventions | treatment | Not Available | completed |
| NCT00744692
| Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders | | treatment | 1 | completed |
| NCT06490601
| Long Term Beta Thalassemia Treatment: Findings From The Extension Period | | treatment | 2 | active_not_recruiting |
| NCT02231710
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases | | treatment | 1 | terminated |
| NCT03609814
| Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT) | | Not Available | Not Available | completed |
| NCT01850108
| Non-Myeloablative Conditioning and Bone Marrow Transplantation | | treatment | Not Available | active_not_recruiting |
| NCT00673608
| Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload | | treatment | 4 | completed |