26 completed, 9 unknown, 4 recruiting, 1 active not recruiting. 43 Phase 3, 20 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 43 | Completed 26, Unknown 9, Recruiting 4, Active not recruiting 1, Enrolling by invitation 1, Not yet recruiting 1, Terminated 1 |
| Phase 2 | 20 | Completed 12, Unknown 8 |
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 |
|---|---|---|---|---|---|---|
| Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia Thalidomide, Danazol, Prednisolone - Beta-Thalassemia, Thalassemia | Phase 3 | Recruiting | Khyber Medical University Peshawar | 172 | 2026-10-12 | 2026-10-21 |
| Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia Iron chelating intervention, N Acetyl cysteine 600mg, Alpha Lipoic Acid 600 MG Oral Tablets - Beta Thalassemia | Phase 3 | Not yet recruiting | Tanta University | 66 | 2025-08-30 | 2025-09-05 |
| A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia Etavopivat A, Etavopivat B, Etavopivat C - Sickle Cell Disease, Thalassemia | Phase 3 | Recruiting | Novo Nordisk A/S | 480 | 2025-01-10 | 2026-09-18 |
| 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 |
| Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease CTX001 - Beta-Thalassemia, Thalassemia | Phase 3 | Recruiting | Vertex Pharmaceuticals Incorporated | 26 | 2022-08-02 | 2026-03-23 |
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) CTX001 - Beta-Thalassemia, Thalassemia | Phase 3 | Active not recruiting | Vertex Pharmaceuticals Incorporated | 16 | 2022-05-03 | 2026-07-01 |
| A Long-term Follow-up Study in Participants Who Received CTX001 CTX001 - Beta-Thalassemia, Thalassemia | Phase 3 | Enrolling by invitation | Vertex Pharmaceuticals Incorporated | 160 | 2021-01-20 | 2026-08-11 |
| 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 |
| Gum Arabic as Anti-oxidant, Anti-inflammatory and Fetal Hemoglobin Inducing Agent in Sickle Cell Anemia Patients Acacia Senegal extract, Pectin - Sickle Cell Anemia in Children | Phase 2 / Phase 3 | Unknown | Al-Neelain University | 100 | 2020-02-15 | 2020-01-28 |
| 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 |
| A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials Luspatercept - Myelodysplastic Syndromes (MDS), Beta-thalassemia | Phase 3 | Recruiting | Celgene | 665 | 2019-08-12 | 2026-07-01 |
| 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 |
| the Efficacy and Safety of Sugammadex in Children 0-2 Years Old Sugammadex - Anesthesia, Pediatric Cancer | Phase 2 / Phase 3 | Unknown | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | 62 | 2018-11-15 | 2018-11-02 |
| 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 |
| PK of SOF/LED in HCV - Infected Adolescents With Haematological Disorders Sofosbuvir and Ledipasvir - HCV Infection, Beta Thalassemia Major | Phase 3 | Unknown | Ain Shams University | 24 | 2018-06-11 | 2023-03-06 |
| Denosumab Versus Zoledronic Acid in Thalassemia-Induced Osteoporosis Denosumab 60 MG/ML Prefilled Syringe, Zoledronic Acid 5Mg/Bag 100Ml Inj - Thalassemia Majors (Beta-Thalassemia Major), Osteoporosis | Phase 3 | Terminated | Hamad Medical Corporation | 17 | 2018-05-14 | 2019-12-12 |
| 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 |
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beta-Thalassemia (32) · Iron Overload (8) · Anemia, Sickle Cell (7) · Genetic Diseases, Inborn (4) · Hematologic Diseases (4) · Hemoglobinopathies (4) · Anemia (3) · Anemia, Diamond-Blackfan (3) · Myelodysplastic Syndromes (3) · Hemosiderosis (2) · Diabetes Mellitus (1) · Hemochromatosis (1) · Hepatitis C (1) · Hepatoblastoma (1)
Anemia, Hemolytic (43) · Anemia, Hemolytic, Congenital (43) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (43) · Hemic and Lymphatic Diseases (43) · Anemia (40) · Genetic Diseases, Inborn (39) · Hematologic Diseases (39) · Hemoglobinopathies (39) · Metabolic Diseases (12) · Nutritional and Metabolic Diseases (12) · Iron Metabolism Disorders (9) · Bone Marrow Diseases (5) · Anemia, Aplastic (3) · Anemia, Hypoplastic, Congenital (3)
Deferasirox (8) · Deferoxamine (6) · Deferiprone (5) · Hydroxyurea (5) · Thalidomide (4) · exagamglogene autotemcel (4) · Amlodipine (3) · Isoflurophate (3) · Zoledronic Acid (2) · betibeglogene autotemcel (2) · luspatercept (2) · Acetylcysteine (1) · Alemtuzumab (1) · Antilymphocyte Serum (1) · Ascorbic Acid (1)
Ain Shams University (6) · Novartis Pharmaceuticals (5) · Vertex Pharmaceuticals Incorporated (4) · Celgene (2) · Children's Hospital Karachi (2) · Genetix Biotherapeutics Inc. (2) · University of Campinas, Brazil (2) · Aga Khan University (1) · Al-Neelain University (1) · Beni-Suef University (1) · Blood Diseases Clinic (1) · Carelon Research (1) · Cerus Corporation (1) · Consorzio per Valutazioni Biologiche e Farmacologiche (1) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (1)
Italy (15) · Egypt (13) · United States (13) · United Kingdom (11) · Germany (10) · Greece (8) · Canada (7) · Turkey (Türkiye) (7) · France (6) · Pakistan (6) · Lebanon (5) · Thailand (5) · Belgium (4) · Iran (4) · Saudi Arabia (4)
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) · Anna Konstantinova, MD (1) · Antonio Piga, MD (1) · Aurelio Maggio, MD (1) · Babar Hasan (1) · Basma A Mansour, Lecturer (1) · Behjat Moayedi, Professor (1) · Charles Quinn, MD (1)
PMID 37975597, PMID 40862696, PMID 42252696, PMID 17951527, PMID 18055982, PMID 18351337, PMID 19951979, PMID 19996412, PMID 20424435, PMID 20566896, PMID 21071497, PMID 22180427, PMID 23585526, PMID 23585527, PMID 23927461, PMID 25492271, PMID 25600572, PMID 25691036, PMID 27326168, PMID 27412888, PMID 29119631, PMID 30504333, PMID 30617198, PMID 31362654, PMID 32212518, PMID 32470438, PMID 32793403, PMID 33283989, PMID 33570654, PMID 33926431, PMID 34175041, PMID 34186262, PMID 34891223, PMID 35477175, PMID 37604857, PMID 37704579, PMID 37782758, PMID 38657265, PMID 39527960, PMID 39947215, PMID 40857358, PMID 41857682, PMID 42274009
2004 (1), U01HL065238 (1)