| Not Yet Recruiting | Evaluation of Micro RNA-155 Expression in Relation to Alloantibody Formation in Transfusion-Dependent Patients NCT07480980 | Assiut University | — |
| Recruiting | Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies NCT06647979 | Daniel Bauer | Phase 1 |
| Not Yet Recruiting | Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia NCT07157722 | Tanta University | Phase 3 |
| Enrolling By Invitation | A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection NCT06717932 | CorrectSequence Therapeutics Co., Ltd | — |
| Recruiting | ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia NCT06364774 | Children's Hospital of Philadelphia | Phase 1 / Phase 2 |
| Enrolling By Invitation | A Long-term Follow-up Study in Participants Who Received CS-101 NCT06685536 | CorrectSequence Therapeutics Co., Ltd | — |
| Recruiting | A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia NCT06772766 | Mabwell (Shanghai) Bioscience Co., Ltd. | Phase 1 |
| Not Yet Recruiting | Plasticizer Exposure and Its Consequences on Health NCT06512298 | Medical University of Vienna | — |
| Recruiting | Adherence of Beta Thalssemia Patients to Oral Chelation Therapy NCT06568926 | Sohag University | N/A |
| Recruiting | A Long-term Follow-up Study in Participants Who Received CS-101 NCT06479616 | Children's Hospital of Fudan University | N/A |
| Recruiting | FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases NCT06539169 | xCures | — |
| Recruiting | An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassem NCT06308159 | Lantu Biopharma | Phase 1 / Phase 2 |
| Not Yet Recruiting | Thrombophilia In Beta Thalassemia NCT04219449 | Assiut University | — |
| Recruiting | A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic S NCT06073860 | Bristol-Myers Squibb | — |
| Enrolling By Invitation | CS-101 in Patients With β-thalassemia NCT06328764 | CorrectSequence Therapeutics Co., Ltd | EARLY_Phase 1 |
| Recruiting | A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel NCT06271512 | Genetix Biotherapeutics Inc. | — |
| Active Not Recruiting | Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major NCT06065189 | Children's Hospital of Fudan University | EARLY_Phase 1 |
| Completed | A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia NCT06024876 | CorrectSequence Therapeutics Co., Ltd | EARLY_Phase 1 |
| Unknown | The Prevelence of HBB c.93-21 G-A in β Thalassemia Patients NCT05133388 | Assiut University | — |
| Enrolling By Invitation | Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia NCT05577312 | Bioray Laboratories | Phase 1 / Phase 2 |
| Active Not Recruiting | A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfus NCT05567458 | Bristol-Myers Squibb | Phase 2 |
| Completed | Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada NCT05506358 | University of British Columbia | N/A |
| 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 |
| Active Not Recruiting | Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Dis NCT05357482 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 1 / Phase 2 |
| Active Not Recruiting | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia NCT05356195 | Vertex Pharmaceuticals Incorporated | Phase 3 |
| Unknown | The Prevelence of IVS 1-6 (T-C) [HBB:c.92 +6 T-C] Gene Mutation in Suspected Cases of β Thalassemia in Assiut NCT05370677 | Assiut University | — |
| 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 |
| Completed | Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta Thalassemia NCT05469230 | Cairo University | — |
| Recruiting | Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Init NCT05635266 | Sanguine Biosciences | — |
| Withdrawn | Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia NCT04938635 | Vifor (International) Inc. | Phase 2 |
| Completed | Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot NCT06239389 | National Institute of Blood and Marrow Transplant (NIBMT), Pakistan | Phase 2 |
| Completed | Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (H NCT04973280 | Celgene | — |
| Completed | Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation NCT06509581 | Ain Shams University | N/A |
| Unknown | Evaluation of Heart Status in Patients of Beta Thalassemia Using Echocardiogram NCT04929574 | Nanfang Hospital, Southern Medical University | — |
| Unknown | Iron Status in BTM With Blood Transfusion NCT04947540 | Assiut University | — |
| Unknown | Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) NCT04247750 | Università degli Studi di Ferrara | 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 |
| Enrolling By Invitation | A Long-term Follow-up Study in Participants Who Received CTX001 NCT04208529 | Vertex Pharmaceuticals Incorporated | Phase 3 |
| Withdrawn | SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias NCT03801889 | Abfero Pharmaceuticals, Inc | Phase 2 |
| Unknown | Complications in Children With B- Thalassemia Major NCT03462784 | Assiut University | — |
| Completed | Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-t NCT04364269 | Vifor (International) Inc. | Phase 2 |
| Completed | ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19 NCT06831799 | Hospital Universitari Vall d'Hebron Research Institute | — |
| Unknown | Dermatological Abnormalities in Beta-thalassemia Major NCT03894605 | Assiut University | — |
| Unknown | Association of Hb F Level With Clinical Severity of Beta Thalassemia NCT04917978 | Nanfang Hospital, Southern Medical University | — |
| Recruiting | Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia NCT04143724 | Celgene | Phase 2 |
| Recruiting | A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Cli NCT04064060 | Celgene | Phase 3 |
| Completed | Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients NCT03877809 | Rare Partners srl Impresa Sociale | Phase 2 |
| Completed | Choroidal Thickness in Beta-thalassemia Patients NCT04067258 | University Hospital of Patras | — |
| Unknown | Detection Of β-thalassemia Carriers In Assiut NCT03822585 | Assiut University | N/A |
| Completed | Resistance Exercise on Postprandial Hyperglycemia in Patients With B-thalassemia Exhibiting Resistance to Insu NCT03889977 | University of Thessaly | N/A |
| Unknown | the Efficacy and Safety of Sugammadex in Children 0-2 Years Old NCT03728543 | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | Phase 2 / Phase 3 |
| Enrolling By Invitation | Early Check: Expanded Screening in Newborns NCT03655223 | RTI International | — |
| Completed | A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia NCT03655678 | Vertex Pharmaceuticals Incorporated | Phase 2 / Phase 3 |
| Unknown | Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector NCT03351829 | Shenzhen Geno-Immune Medical Institute | N/A |
| Terminated | A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Bet NCT03381833 | La Jolla Pharmaceutical Company | Phase 2 |
| Completed | A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia NCT03271541 | Hoffmann-La Roche | Phase 2 |
| Completed | Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus NCT03961828 | Tanta University | Phase 4 |
| Completed | A Study of EPEG in Beta Thalassemia Patients NCT02950857 | Prolong Pharmaceuticals | Phase 1 |
| Completed | A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfu NCT03207009 | Genetix Biotherapeutics Inc. | Phase 3 |
| Unknown | Genetic Variants Affecting the Clinical Severity of Beta Thalassemia NCT04918056 | Nanfang Hospital, Southern Medical University | — |
| Completed | A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfu NCT02906202 | Genetix Biotherapeutics Inc. | Phase 3 |
| Completed | An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood NCT02604433 | Celgene | Phase 3 |
| Completed | To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β- NCT02626689 | Celgene | — |
| Unknown | Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia Pati NCT02474420 | Kevin H.M. Kuo, MD, MSc, FRCPC | N/A |
| Unknown | Gene Therapy for Transfusion Dependent Beta-thalassemia NCT02453477 | IRCCS San Raffaele | Phase 1 / Phase 2 |
| Unknown | Thalassemia Treatment Based on the Stem Cell Technology NCT03222453 | Xiaofang Sun | N/A |
| Withdrawn | Treatment of Iron Overload Requiring Chelation Therapy NCT01927913 | Shire | Phase 2 |
| Unknown | Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia NCT01996683 | Ain Shams University | N/A |
| Completed | Effects of Spirulina on Cardiac Functions in Children With Beta Thalassemia Major NCT02597595 | Tanta University | N/A |
| Terminated | Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassem NCT02274233 | Sideris Pharmaceuticals | Phase 1 |
| Completed | At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurolo NCT01931644 | Sanguine Biosciences | — |
| Completed | CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant NCT01049854 | New York Medical College | Phase 2 |
| Terminated | Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iro NCT01363908 | Shire | Phase 2 |
| Withdrawn | Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies NCT01419704 | Talaris Therapeutics Inc. | Phase 1 / Phase 2 |
| Completed | Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major NCT01206075 | University of Washington | N/A |
| Completed | Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload NCT01186419 | Shire | Phase 2 |
| Unknown | Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patient NCT01016093 | Tehran University of Medical Sciences | Phase 2 / Phase 3 |
| Completed | Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overlo NCT01039636 | FerroKin BioSciences, Inc. | Phase 1 |
| Completed | Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia NCT00790127 | HemaQuest Pharmaceuticals Inc. | Phase 1 / Phase 2 |
| Completed | Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Be NCT00564941 | Novartis Pharmaceuticals | Phase 4 |
| Completed | Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major NCT00336362 | University of Washington | N/A |
| Completed | Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment NCT00447694 | Novartis | Phase 2 |
| Terminated | Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases NCT00115349 | Carelon Research | Phase 2 |
| Active Not Recruiting | Stem Cell Transplant in Sickle Cell Disease and Thalassemia NCT00408447 | Columbia University | Phase 2 |
| Completed | Genetic Factors Affecting the Severity of Beta Thalassemia NCT00159042 | Deborah Rund | — |
| Unknown | Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major NCT00103753 | Royal Brompton & Harefield NHS Foundation Trust | Phase 4 |
| Completed | A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosidero NCT00171171 | Novartis | Phase 3 |
| Completed | Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalasse NCT00069862 | Genzyme, a Sanofi Company | Phase 1 / Phase 2 |
| Completed | Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Tran NCT00061750 | Novartis Pharmaceuticals | Phase 3 |
| Completed | Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic NCT00061763 | Novartis Pharmaceuticals | Phase 2 |
| Completed | Efficacy Study of the Use of Sequential DFP-DFO Versus DFP NCT00733811 | Azienda Ospedaliera V. Cervello | Phase 4 |
| Completed | Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN) NCT00000623 | Carelon Research | — |
| Completed | 5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia NCT00005934 | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Phase 2 |
| Completed | Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia) NCT00001958 | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Phase 2 |
| Completed | Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia NCT00006136 | Boston University | Phase 2 |
| Completed | Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia NCT00029380 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Completed | Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone NCT00000588 | Case Western Reserve University | Phase 2 |
| Completed | Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis NCT00000595 | National Heart, Lung, and Blood Institute (NHLBI) | Phase 2 |
| Unknown | Prevalence Of Microalbuminuria Among Children Suffering From Sickle Cell Nephropathy and Sickle Cell/Beta-Thal NCT01092169 | Hillel Yaffe Medical Center | — |