26 completed. 26 Phase 3, 12 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 26 | Completed 26 |
| Phase 2 | 12 | Completed 12 |
Follow Thalassemia: new trials by email Literature Review of Thalassemia trials Research Report on Thalassemia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Effect of L-Glutamine on Pulmonary Artery Pressure in Patients With Non-Transfusion-Dependent Thalassemia L-glutamine - Pulmonary Artery Pressure, Thalassemia | Phase 2 / Phase 3 | Completed | Mazandaran University of Medical Sciences | 8 | 2023-08-15 | 2025-10-07 |
| Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients Hydroxyurea and Thalidomide - Thalassemia, Beta | Phase 2 / Phase 3 | Completed | Children's Hospital Karachi | 603 | 2020-07-07 | 2023-12-01 |
| Clinical Experience of Thalidomide in Thalassemic Patients hydroxyurea and thalidomide - Thalassemia, Beta | Phase 2 / Phase 3 | Completed | Children's Hospital Karachi | 135 | 2020-01-01 | 2021-11-24 |
| Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia Omega 3, Nigella Sativa Oil, Hydroxyurea - Iron Overload, Oxidative Stress | Phase 2 / Phase 3 | Completed | Beni-Suef University | 350 | 2019-11-01 | 2021-01-27 |
| Evaluate the Efficacy and Safety of Irochel for the Management of Iron Overload in Thalassemia Patients in Comparison With Deferoxamine Irochel, Deferoxamine - Iron Overload Due to Repeated Red Blood Cell Transfusions, Thalassemia Majors (Beta-Thalassemia Major) | Phase 3 | Completed | Hamdard University | 132 | 2019-02-11 | 2026-08-25 |
| A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia CTX001 - Beta-Thalassemia, Thalassemia | Phase 2 / Phase 3 | Completed | Vertex Pharmaceuticals Incorporated | 59 | 2018-09-14 | 2025-12-17 |
| Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Thalidomide and Hydroxyurea - Thalassemia | Phase 2 / Phase 3 | Completed | Blood Diseases Clinic | 654 | 2018-01-01 | 2022-03-25 |
| Zinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes Mellitus Zinc - Beta-thalassemia Major Complicated With Diabetes | Phase 3 | Completed | Ain Shams University | 80 | 2017-08-01 | 2019-02-25 |
| A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia LentiGlobin BB305 Drug Product - Beta-Thalassemia | Phase 3 | Completed | Genetix Biotherapeutics Inc. | 19 | 2017-06-08 | 2024-03-07 |
| Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation Deferasirox - Chronic Iron Overload | Phase 3 | Completed | Novartis Pharmaceuticals | 53 | 2016-08-16 | 2020-03-03 |
| A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype LentiGlobin BB305 Drug Product - Beta-Thalassemia | Phase 3 | Completed | Genetix Biotherapeutics Inc. | 24 | 2016-08-08 | 2023-06-18 |
| An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia Luspatercept, Placebo - Erythrocyte Transfusion, Beta-Thalassemia | Phase 3 | Completed | Celgene | 336 | 2016-05-02 | 2023-04-18 |
| Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients Deferiprone, Deferasirox - Chronic Iron Overload | Phase 3 | Completed | Consorzio per Valutazioni Biologiche e Farmacologiche | 435 | 2014-03-17 | 2021-05-04 |
| Amlodipine for Myocardial Iron in Thalassemia Standard Chelation, Amlodipine - Thalassemia | Phase 2 / Phase 3 | Completed | Aga Khan University | 20 | 2014-02-01 | 2017-06-29 |
| Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion S-303 Treated Red Blood Cells (RBCs), Conventional, untreated Red Blood Cells - Thalassemia Major | Phase 3 | Completed | Cerus Corporation | 86 | 2012-12-01 | 2018-07-18 |
| Amlodipine in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major Amlodipine - Thalassemia, Iron Overload | Phase 3 | Completed | University of Campinas, Brazil | 62 | 2012-08-01 | 2015-09-09 |
| Effect of Metoprolol on Thalassemia Cardiomyopathy metoprolol, placebo - B Thalassemia | Phase 2 / Phase 3 | Completed | Shiraz University of Medical Sciences | 45 | 2012-01-01 | 2013-05-27 |
| Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension Sildenafil - Thalassemia, Hypertension, Pulmonary | Phase 2 / Phase 3 | Completed | Carelon Research | 27 | 2009-03-01 | 2014-02-21 |
| Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major Amlodipine - Thalassemia Major | Phase 2 / Phase 3 | Completed | University of Campinas, Brazil | 10 | 2008-09-01 | 2015-01-05 |
| Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload Deferasirox - Thalassemia, Sickle Cell Disease | Phase 3 | Completed | Novartis Pharmaceuticals | 1683 | 2005-10-01 | 2011-06-07 |
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beta-Thalassemia (16) · Iron Overload (7) · Anemia (3) · Anemia, Diamond-Blackfan (3) · Anemia, Sickle Cell (2) · Hemosiderosis (2) · Myelodysplastic Syndromes (2) · Diabetes Mellitus (1) · Genetic Diseases, Inborn (1) · Hematologic Diseases (1) · Hemochromatosis (1) · Hemoglobinopathies (1) · Hypertension, Pulmonary (1) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (1)
Anemia, Hemolytic (26) · Anemia, Hemolytic, Congenital (26) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (26) · Hemic and Lymphatic Diseases (26) · Genetic Diseases, Inborn (25) · Hematologic Diseases (25) · Hemoglobinopathies (25) · Anemia (23) · Metabolic Diseases (10) · Nutritional and Metabolic Diseases (10) · Iron Metabolism Disorders (8) · Bone Marrow Diseases (4) · Anemia, Aplastic (3) · Anemia, Hypoplastic, Congenital (3)
Deferasirox (7) · Deferoxamine (5) · Hydroxyurea (4) · Amlodipine (3) · Thalidomide (3) · Deferiprone (2) · betibeglogene autotemcel (2) · Alemtuzumab (1) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Congresses as Topic (1) · Cyclophosphamide (1) · Docosahexaenoic Acids (1) · Glutamine (1)
Novartis Pharmaceuticals (5) · Children's Hospital Karachi (2) · Genetix Biotherapeutics Inc. (2) · University of Campinas, Brazil (2) · Aga Khan University (1) · Ain Shams University (1) · Beni-Suef University (1) · Blood Diseases Clinic (1) · Carelon Research (1) · Celgene (1) · Cerus Corporation (1) · Consorzio per Valutazioni Biologiche e Farmacologiche (1) · Hamdard University (1) · Lipomed (1) · Masonic Cancer Center, University of Minnesota (1)
Italy (10) · United States (8) · United Kingdom (7) · Egypt (6) · Greece (6) · Germany (5) · Pakistan (5) · Turkey (Türkiye) (5) · France (4) · Thailand (4) · Canada (3) · Lebanon (3) · Taiwan (3) · Australia (2) · Austria (2)
Ahmed F Mahmoud Hussein, MS.c (1) · Ali Taher, Ass.Prof. (1) · Ali Taher, MD (1) · Amal M El-Beshlawy, Prof. Dr. (1) · Angela Smith, MD (1) · Antonio Piga, MD (1) · Aurelio Maggio, MD (1) · Babar Hasan (1) · Charles Quinn, MD (1) · Claudia Morris, MD (1) · Donato Bonifazi, Dr (1) · Ellis Neufeld, MD, PhD (1)
PMID 37975597, PMID 17951527, PMID 18055982, PMID 18351337, PMID 19951979, PMID 19996412, PMID 20424435, PMID 20566896, PMID 21071497, PMID 22180427, PMID 23585526, PMID 23585527, PMID 25492271, PMID 25691036, PMID 27326168, PMID 27412888, PMID 30504333, PMID 30617198, PMID 31362654, PMID 32212518, PMID 32470438, PMID 32793403, PMID 33283989, PMID 33570654, PMID 33926431, PMID 34175041, PMID 34891223, PMID 35477175, PMID 37604857, PMID 37704579, PMID 37782758, PMID 38657265, PMID 39527960, PMID 39947215, PMID 40862696, PMID 42252696
2004 (1), U01HL065238 (1)