33 completed. 33 Phase 1, 11 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 33 | Completed 33 |
| Phase 2 | 11 | Completed 11 |
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 |
|---|---|---|---|---|---|---|
| A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants Etavopivat, Moxifloxacin, Placebo - Healthy Volunteers, Sickle Cell Disease | Phase 1 | Completed | Novo Nordisk A/S | 33 | 2025-06-09 | 2026-01-02 |
| A Research Study of the Effect of Etavopivat on Other Drugs in Healthy Participants Etavopivat, Digoxin, Pitavastatin - Healthy Volunteers Sickle Cell Disease, Thalassemia | Phase 1 | Completed | Novo Nordisk A/S | 37 | 2025-02-06 | 2026-09-29 |
| A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants Etavopivat - Healthy Volunteers, Sickle Cell Disease | Phase 1 | Completed | Novo Nordisk A/S | 24 | 2024-08-26 | 2025-12-29 |
| A Research Study of the Effect of Food on Etavopivat in Healthy Participants Etavopivat - Healthy Volunteers Sickle Cell Disease, Thalassemia | Phase 1 | Completed | Novo Nordisk A/S | 16 | 2024-05-28 | 2025-10-10 |
| A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major CS-101 injection - Beta-Thalassemia Major | Phase 1 | Completed | CorrectSequence Therapeutics Co., Ltd | 9 | 2024-04-18 | 2026-02-10 |
| Safety & Efficacy of Thalidomide in Children With Transfusion Dependent Thalassemia Thalidomide Capsules, Placebo - Transfusion-dependent Thalassemia | Phase 1 | Completed | Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh | 60 | 2023-03-01 | 2024-04-09 |
| The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major N-acetyl cysteine - Thalassemia Major | Phase 1 | Completed | Beni-Suef University | 100 | 2019-06-03 | 2020-02-07 |
| Pharmacokinetics of Oral Hydroxyurea Solution Oral Hydroxyurea (100 mg/mL) Solution - Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia | Phase 1 / Phase 2 | Completed | Nova Laboratories Limited | 33 | 2019-01-03 | 2024-10-28 |
| Short-term Clinical Study of CN128 in Thalassemia Patients CN128 Tablets - Thalassemia | Phase 1 | Completed | Hangzhou Zede Pharma-Tech Co., Ltd. | 16 | 2018-12-03 | 2023-06-09 |
| Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Thiotepa--single daily dose, Thiotepa--escalated dose - Bone Marrow Failure Syndrome, Thalassemia | Phase 1 / Phase 2 | Completed | University of Florida | 6 | 2018-06-15 | 2023-09-15 |
| A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT) ST-400 Investigational product - Transfusion Dependent Beta-thalassemia | Phase 1 / Phase 2 | Completed | Sangamo Therapeutics | 5 | 2018-03-29 | 2023-12-14 |
| Clinical Study of CN128 in Thalassemia Patients CN128, placebo - Thalassemia | Phase 1 | Completed | Hangzhou Zede Pharma-Tech Co., Ltd. | 32 | 2018-01-05 | 2019-12-19 |
| A Study of EPEG in Beta Thalassemia Patients EPEG - Beta-Thalassemia | Phase 1 | Completed | Prolong Pharmaceuticals | 6 | 2017-06-28 | 2018-05-25 |
| Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers IONIS TMPRSS6-Lrx, Placebo - Thalassemia | Phase 1 | Completed | Ionis Pharmaceuticals, Inc. | 36 | 2017-05-09 | 2019-01-22 |
| Haploidentical Hematopoietic Stem Cell Transplantation peripheral blood stem cell graft that are CD34+ selected - Sickle Cell-thalassemia Disease, Thalassemia | Phase 1 | Completed | Catherine Bollard | 7 | 2015-01-01 | 2024-12-19 |
| Bone Marrow Transplant With Abatacept for Non-Malignant Diseases Abatacept - Hurler Syndrome, Fanconi Anemia | Phase 1 | Completed | Emory University | 10 | 2014-01-01 | 2019-12-26 |
| A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease LentiGlobin BB305 Drug Product - Beta-Thalassemia Major, Sickle Cell Disease | Phase 1 / Phase 2 | Completed | Genetix Biotherapeutics Inc. | 7 | 2013-06-07 | 2022-04-26 |
| Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies NiCord - Sickle Cell Disease & Thalassemia | Phase 1 / Phase 2 | Completed | Gamida Cell ltd | 16 | 2012-09-01 | 2022-05-20 |
| Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia Deferasirox and deferiprone - Thalassemia Major With Severe Transfusional Iron Overload | Phase 1 / Phase 2 | Completed | Children's Hospital of Philadelphia | 9 | 2012-09-01 | 2019-03-06 |
| Immunomodulatory Effects of Silymarin in Patients With Beta-Thalassemia Major Desferrioxamine, Legalon® (Silymarin), Silymarin (Legalon) - Immune Abnormalities | Phase 1 | Completed | Shiraz University of Medical Sciences | 25 | 2012-06-01 | 2012-12-19 |
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Anemia, Sickle Cell (14) · beta-Thalassemia (14) · Mucopolysaccharidosis I (4) · Anemia, Diamond-Blackfan (3) · Granulomatous Disease, Chronic (3) · Iron Overload (3) · Wiskott-Aldrich Syndrome (3) · Anemia (2) · Anemia, Aplastic (2) · Chediak-Higashi Syndrome (2) · Genetic Diseases, Inborn (2) · Hemoglobin SC Disease (2) · Hemoglobinopathies (2) · Lymphohistiocytosis, Hemophagocytic (2)
Anemia, Hemolytic, Congenital (33) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (33) · Hematologic Diseases (33) · Hemic and Lymphatic Diseases (33) · Anemia, Hemolytic (32) · Anemia (31) · Genetic Diseases, Inborn (31) · Hemoglobinopathies (31) · Metabolic Diseases (7) · Nutritional and Metabolic Diseases (7) · Skin and Connective Tissue Diseases (5) · Bone Marrow Diseases (4) · Carbohydrate Metabolism, Inborn Errors (4) · Connective Tissue Diseases (4)
Alemtuzumab (2) · Hematopoietic Stem Cell Transplantation (2) · Hydroxyurea (2) · Melphalan (2) · fludarabine (2) · 2,2-dimethylbutyric acid (1) · 4'-hydroxydesazadesferrithiocin (1) · 4,5-dihydro-2-(2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl)-4-methyl-4-thiazolecarboxylic acid (1) · Abatacept (1) · Acetylcysteine (1) · Cyclosporine (1) · Deferasirox (1) · Deferiprone (1) · Deferoxamine (1) · Digoxin (1)
Novo Nordisk A/S (4) · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) (3) · Hangzhou Zede Pharma-Tech Co., Ltd. (2) · National Heart, Lung, and Blood Institute (NHLBI) (2) · Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh (1) · Beni-Suef University (1) · Catherine Bollard (1) · Celgene (1) · Children's Hospital Los Angeles (1) · Children's Hospital of Philadelphia (1) · CorrectSequence Therapeutics Co., Ltd (1) · Duke University (1) · Emory University (1) · FerroKin BioSciences, Inc. (1) · Gamida Cell ltd (1)
United States (20) · China (4) · Thailand (3) · Australia (2) · Bangladesh (1) · Egypt (1) · France (1) · Iran (1) · Jamaica (1) · Lebanon (1) · Singapore (1) · United Kingdom (1)
Angela E Rankine- Mullings, MD (1) · Biljana Horn, MD (1) · Ellis Neufeld, MD (1) · Heba M Ahmed (1) · Hisham Abdel-Azim, MD (1) · Janet L Kwiatkowski, MD, MSCE (1) · Jean-Antoine Ribeil, MD (1) · Jianzhong Shentu (1) · Joanne Kurtzberg, MD (1) · Joel Brochstein, MD (1) · John F Tisdale, M.D. (1) · John T Horan, MD (1) · Matthew M Hsieh, M.D. (1)
PMID 17536019, PMID 17620452, PMID 18439858, PMID 21742281, PMID 24296492, PMID 24916507, PMID 25222041, PMID 25938131, PMID 26348869, PMID 26589907, PMID 28249145, PMID 29455932, PMID 29669226, PMID 33560399, PMID 34175041, PMID 34795208, PMID 35075288, PMID 35745672, PMID 36058507, PMID 36240296, PMID 36637054, PMID 36690839, PMID 37975597
2017 (1), R01DK055463 (1)