| Not Yet Recruiting | A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major NCT07489196 | CorrectSequence Therapeutics Co., Ltd | Phase 2 |
| Recruiting | Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-tha NCT07112703 | Rawan Mohammed Khairy Mostafa Elsawy | N/A |
| Recruiting | Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia NCT06931912 | Institute of Hematology & Blood Diseases Hospital, China | N/A |
| Active Not Recruiting | A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan NCT06596642 | Bristol-Myers Squibb | — |
| Recruiting | Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia NCT06490627 | National Institute of Blood and Marrow Transplant (NIBMT), Pakistan | Phase 2 |
| Unknown | NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major NCT05777733 | Assiut University | Phase 1 |
| Suspended | Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells NCT05442346 | Bioray Laboratories | N/A |
| Unknown | Longitudinal Study on the QoL of Pediatric Patients After HSCT and Its Influencing Factors NCT06026839 | Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | — |
| Recruiting | Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells NCT06041620 | Institute of Hematology & Blood Diseases Hospital, China | N/A |
| Recruiting | Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thal NCT05776173 | Shanghai BDgene Co., Ltd. | N/A |
| Active Not Recruiting | Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dos NCT06913634 | Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia | — |
| Active Not Recruiting | To Evaluate Long- Term Safety and Efficacy of Luspatercept NCT06875219 | Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia | — |
| Unknown | Study on the Effect of Different Risk Factors on the Growth Parameters of Thalassemic Patients in Assiut (AUCH NCT05303506 | Assiut University | — |
| Unknown | The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-w NCT05462548 | Sun Yat-sen University | Phase 4 |
| 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 |
| Terminated | β-globin Restored Autologous HSC in β-thalassemia Major Patients NCT04205435 | Bioray Laboratories | Phase 1 / Phase 2 |
| Completed | Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells NCT04211480 | Bioray Laboratories | N/A |
| Completed | Frequency of COVID-19 Antibodies in Patients With Hereditary Hematological Diseases NCT04526405 | University of Milano Bicocca | — |
| Unknown | Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation NCT04291352 | University Health Network, Toronto | N/A |
| Active Not Recruiting | Pilot Study PBSCT with TCRab Depletion for Hemoglobinopathies NCT04523376 | Timothy Olson | N/A |
| Completed | Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalasse NCT04292314 | Beni-Suef University | Phase 2 / Phase 3 |
| Completed | Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Pati NCT03637556 | DisperSol Technologies, LLC | Phase 2 |
| Completed | Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinic NCT04009525 | First Affiliated Hospital of Guangxi Medical University | Phase 4 |
| Completed | The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major NCT04260516 | Beni-Suef University | Phase 1 |
| Unknown | Observing the Changes of Endocrine and Metabolism in Patients With Thalassemia Major NCT03951818 | National Taiwan University Hospital | — |
| Completed | Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital NCT03161899 | Assiut University | — |
| Completed | Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients NCT03992001 | Università degli Studi di Ferrara | Phase 4 |
| Terminated | In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM) NCT02986698 | University of California, San Francisco | Phase 1 |
| Unknown | Precision Exercise in Children With Malignant Hemopathies NCT04090268 | University of Milano Bicocca | N/A |
| Unknown | Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major NCT03171831 | First Affiliated Hospital of Guangxi Medical University | Phase 4 |
| Completed | Periodontal Therapy in Thalassemia Major Patients NCT03311243 | King Saud University | N/A |
| Completed | Denosumab vs Placebo in Patients With Thalassemia Major and Osteoporosis NCT02559648 | Ersi Voskaridou | Phase 2 |
| Completed | Long Term Outcomes in β Thalassemia Major NCT02307786 | Ann & Robert H Lurie Children's Hospital of Chicago | — |
| Completed | Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia. NCT02049450 | Novartis Pharmaceuticals | Phase 2 |
| Unknown | Role of Vitamin C to Augment Iron Chelation With DFP or DFX NCT02083575 | Ain Shams University | Phase 2 / Phase 3 |
| Completed | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases NCT01917708 | Emory University | Phase 1 |
| Completed | A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Partic NCT01745120 | Genetix Biotherapeutics Inc. | Phase 1 / Phase 2 |
| Completed | MR Imaging of Diffuse Myocardial Fibrosis in Transfusion-Dependent Anemias NCT02090699 | University Health Network, Toronto | — |
| Unknown | "Iron Overload and Endocrinological Diseases" NCT06137079 | Azienda Ospedaliero-Universitaria di Modena | — |
| Withdrawn | An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Adminis NCT01724138 | Novartis Pharmaceuticals | Phase 4 |
| Completed | Reproductive Capacity and Iron Burden in Thalassemia NCT02308904 | UCSF Benioff Children's Hospital Oakland | N/A |
| Completed | Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major NCT01740531 | Cerus Corporation | Phase 3 |
| Unknown | Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patient NCT02126046 | Nanfang Hospital, Southern Medical University | N/A |
| Completed | Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia NCT01496963 | Ente Ospedaliero Ospedali Galliera | — |
| Unknown | The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study NCT01323608 | Weill Medical College of Cornell University | Phase 4 |
| Completed | High-Tc Susceptometer to Monitor Transfusional Iron Overload NCT01241357 | Columbia University | — |
| Unknown | B Memory Cell Response to Vaccination With the 13-valent Pneumococcal Conjugate Vaccine in Asplenic Individual NCT01846923 | Aghia Sophia Children's Hospital of Athens | Phase 4 |
| Completed | Perceptions of Thalassemia Major in Singapore: An Exploratory Study of Stigma NCT01129076 | National Human Genome Research Institute (NHGRI) | — |
| Completed | Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major NCT01125254 | University of Campinas, Brazil | Phase 2 / Phase 3 |
| Completed | Pilot Study for Patients With Poor Response to Deferasirox NCT00749515 | Boston Children's Hospital | Phase 4 |
| Completed | Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thal NCT00480506 | Dana-Farber Cancer Institute | — |
| Completed | Efficacy and Safety of Neridronate (Nerixia®)to Treat Osteoporosis in Patients With TM and TI NCT01140321 | Ente Ospedaliero Ospedali Galliera | Phase 2 |
| Completed | A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Majo NCT00390858 | Novartis Pharmaceuticals | Phase 2 |
| Completed | Efficacy Study in Removing Excess Iron From the Heart NCT00105495 | ApoPharma | Phase 4 |
| 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 | Efficacy Study of the Use of Sequential DFP-DFO Versus DFP NCT00733811 | Azienda Ospedaliera V. Cervello | Phase 4 |
| Completed | Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant He NCT00005893 | Fairview University Medical Center | N/A |
| Completed | Diagnostic Pilot Study of Dual Energy Absorptiometry in the Detection of Osteopenia or Osteoporosis in Patient NCT00006138 | National Center for Research Resources (NCRR) | — |
| Completed | Stem Cell Transplantation (SCT) for Genetic Diseases NCT00004378 | National Center for Research Resources (NCRR) | N/A |