Thalassemia: Clinical Trials

241 completed, 75 unknown, 42 recruiting, 31 terminated. 138 Phase 2, 81 Not applicable, 64 Phase 1, 43 Phase 3, 31 Phase 4.

By phase and status

Phase	Trials	Status
Phase 2	138	Completed 69, Terminated 18, Unknown 16, Active not recruiting 15, Recruiting 9, Withdrawn 7, Not yet recruiting 3, Enrolling by invitation 1
Not applicable	81	Completed 42, Unknown 17, Recruiting 6, Active not recruiting 5, Terminated 5, Not yet recruiting 4, Enrolling by invitation 1, Suspended 1
Phase 1	64	Completed 33, Active not recruiting 9, Terminated 8, Recruiting 7, Unknown 5, Enrolling by invitation 1, Withdrawn 1
Phase 3	43	Completed 26, Unknown 9, Recruiting 4, Active not recruiting 1, Enrolling by invitation 1, Not yet recruiting 1, Terminated 1
Phase 4	31	Completed 19, Unknown 8, Active not recruiting 2, Enrolling by invitation 1, Withdrawn 1
Early Phase 1	9	Recruiting 3, Completed 2, Unknown 2, Enrolling by invitation 1, Withdrawn 1

Follow Thalassemia: new trials by email Literature Review of Thalassemia trials Research Report on Thalassemia

Trials newest first · largest first

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
Heterologous Cord Blood-Derived Red Blood Cell for Transfusion in Extremely Preterm Infants Cord Blood Red Blood Cells, Standard Adult Donor Red Blood Cells - Extremely Premature Infant, Anemia Neonatal	Not applicable	Not yet recruiting	Hospital Clinic of Barcelona	176	2027-01-01	2026-03-31
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector Gene-modified autologous hematopoeitic stem cells - Beta-Thalassemia	Not applicable	Not yet recruiting	Shenzhen Geno-Immune Medical Institute	20	2026-12-31	2026-08-27
The Effect of Motivational Interviewing on Treatment Adherence and Self-Efficacy Among Omani Adults With Thalassemia Motivational Interviewing - Beta-Thalassemia Major, Transfusion-Dependent Thalassemia	Not applicable	Not yet recruiting	Oman Ministry of Health	45	2026-11-01	2026-06-22
Native T1 Mapping and ECV in Chronic Hemolytic Anemia Hemolytic Anemia, Thalassemia		Not yet recruiting	Assiut University	54	2026-10-20	2026-10-20
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
Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia HGI-001 Injection - Beta Thalassemia Transfusion Dependent	Phase 2	Not yet recruiting	Prince of Wales Hospital, Shatin, Hong Kong	6	2026-09-01	2026-07-23
Clinical, Economic,and Psychosocial Burden of Thalassemia in Sohag Thalassemia Particularly Transfusion Dependent Thalassemia		Not yet recruiting	Randa Elham Hamam	100	2026-08-01	2026-08-31
Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors Total Body Irradiation, Alemtuzumab, Abatacept - Sickle Cell Disease, Beta-thalassemia	Phase 1 / Phase 2	Recruiting	National Heart, Lung, and Blood Institute (NHLBI)	90	2026-07-08	2026-10-13
Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation Intravenous Immunoglobulin, Dexamethasone, Mononuclear Cells - Leukemia, Lymphoma	Phase 2	Not yet recruiting	Hematology department of the 920th hospital	60	2026-07-03	2026-07-14
Evaluation of Micro RNA-155 Expression in Relation to Alloantibody Formation in Transfusion-Dependent Patients Red Cell Alloantibodies, Blood Transfusion-Dependent		Not yet recruiting	Assiut University	100	2026-07-01	2026-04-06
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia Thalidomide, Hydroxy Urea - Thalassemia	Not applicable	Recruiting	Riphah International University	240	2026-06-20	2026-08-06
A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT) SNH-119014 dose 1, SNH-119014 dose 2, Placebo - Thalassemia	Phase 2	Not yet recruiting	ScinnoHub Pharmaceutical Co., Ltd.	30	2026-06-18	2026-05-12
Evaluation of the Quality of Life in Patients With Chronic Iron Overload Due to Hemoglobinopathies in Greece. Thalassemia, Iron Overload		Not yet recruiting	Elpen Pharmaceutical Co. Inc.	150	2026-04-15	2026-01-20
Risk Factors Affecting Growth in Thalassemic Children at AUCH Non-interventional assessment of growth and risk factors in thalassemic children - Thalassemia Majors (Beta-Thalassemia Major)		Not yet recruiting	Assiut University	85	2026-04-01	2026-04-03
Open-Label Extension Study to Pioneer Study 6058-SCD-101 Pociredir - Sickle Cell Disease	Phase 2	Terminated	Fulcrum Therapeutics	1	2026-03-30	2026-10-28
Low-Dose Liposomal Amphotericin B for Invasive Fungal Infection Prophylaxis in Neutropenic Children Liposomal Amphotericin B (LAmB) - Thalassemia, Hematopoietic Stem Cell Transplantation		Recruiting	Haikou Affiliated Hospital of Central South University Xiangya School of Medicine	30	2026-02-01	2026-07-24
Effect of Omega 3 Fatty Acids on Heart and Liver in Beta Thalassemia Patients Omega 3 Fish Oil supplements, Deferasirox - Thalassemia Majors (Beta-Thalassemia Major)	Phase 2	Completed	Ain Shams University	100	2026-01-01	2026-10-07
Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease Pociredir - Sickle Cell Disease	Phase 1	Terminated	Fulcrum Therapeutics	13	2025-12-13	2026-06-18
Transfusion-Related Changes in Oxidative Stress Biomarkers in Neonates Peroxide analysis-TOC Omnignostica Forschungs GmbH, Near Infrared Imaging - Oxidative Stress in Neonates, Near Infrared Spectroscopy		Recruiting	Medical University of Graz	170	2025-12-12	2026-06-26
Gonadal Function in Thalassemic Patient serum inhibin B serum INSL3 serum AMH - Thalassemia Majors (Beta-Thalassemia Major)		Not yet recruiting	Sohag University	80	2025-12-07	2025-12-17

Showing 20 of 457. Sign in to see all of them.

Narrower conditions

beta-Thalassemia (264)  ·  Anemia, Sickle Cell (126)  ·  Iron Overload (55)  ·  Hemoglobinopathies (43)  ·  Anemia, Diamond-Blackfan (30)  ·  Anemia (24)  ·  Hematologic Diseases (21)  ·  Myelodysplastic Syndromes (20)  ·  Genetic Diseases, Inborn (18)  ·  alpha-Thalassemia (16)  ·  Anemia, Aplastic (14)  ·  Fanconi Anemia (12)  ·  Granulomatous Disease, Chronic (11)  ·  Hematologic Neoplasms (11)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (457)  ·  Hemic and Lymphatic Diseases (457)  ·  Anemia, Hemolytic, Congenital (446)  ·  Anemia, Hemolytic (442)  ·  Genetic Diseases, Inborn (439)  ·  Hematologic Diseases (436)  ·  Anemia (426)  ·  Hemoglobinopathies (414)  ·  Nutritional and Metabolic Diseases (102)  ·  Metabolic Diseases (96)  ·  Iron Metabolism Disorders (58)  ·  Bone Marrow Diseases (56)  ·  Pathological Conditions, Signs and Symptoms (54)  ·  Immune System Diseases (48)

Interventions

Deferasirox (33)  ·  Deferoxamine (19)  ·  Hydroxyurea (18)  ·  fludarabine (18)  ·  Cyclophosphamide (17)  ·  Deferiprone (17)  ·  Alemtuzumab (15)  ·  Busulfan (15)  ·  luspatercept (13)  ·  Thalidomide (12)  ·  fludarabine phosphate (12)  ·  Mycophenolic Acid (10)  ·  Tacrolimus (10)  ·  Sirolimus (8)  ·  Melphalan (7)

Sponsors

Novartis Pharmaceuticals (22)  ·  Assiut University (21)  ·  HaEmek Medical Center, Israel (12)  ·  National Heart, Lung, and Blood Institute (NHLBI) (11)  ·  Ain Shams University (10)  ·  Tanta University (9)  ·  University of California, San Francisco (9)  ·  Carelon Research (8)  ·  Celgene (8)  ·  UCSF Benioff Children's Hospital Oakland (6)  ·  Bristol-Myers Squibb (5)  ·  CorrectSequence Therapeutics Co., Ltd (5)  ·  Institute of Hematology & Blood Diseases Hospital, China (5)  ·  Nanfang Hospital, Southern Medical University (5)  ·  National Institute of Allergy and Infectious Diseases (NIAID) (5)

Countries

United States (167)  ·  Italy (60)  ·  China (58)  ·  Egypt (46)  ·  Canada (34)  ·  Thailand (33)  ·  Greece (32)  ·  United Kingdom (30)  ·  Turkey (Türkiye) (29)  ·  Lebanon (26)  ·  Israel (24)  ·  France (17)  ·  Germany (17)  ·  Pakistan (15)  ·  Spain (14)

Top investigators

Ariel Koren, MD (8)  ·  Janet Kwiatkowski, MD (5)  ·  John F Tisdale, M.D. (5)  ·  Carina Levin, MD (4)  ·  Nancy Olivieri, MD (4)  ·  Elliott Vichinsky, MD (3)  ·  John Porter, MD (3)  ·  Patricia J. Giardina, MD (3)  ·  Paul Szabolcs, MD (3)  ·  Petros Kountouris, PhD (3)  ·  Xiaoyang Yang, MD (3)

Results publications

PMID 37975597, PMID 36240296, PMID 42133908, PMID 29931453, PMID 30617198, PMID 33570654, PMID 34175041, PMID 36637054, PMID 37704579, PMID 40010689, PMID 40862696, PMID 41857682, PMID 42233421, PMID 42252696, PMID 42705233, PMID 14550808, PMID 14686498, PMID 16273116, PMID 16737942, PMID 17536019, PMID 17620452, PMID 17951527, PMID 18055982, PMID 18298856, PMID 18351337, PMID 18439858, PMID 18550258, PMID 18568278, PMID 19236376, PMID 19407319, PMID 19724055, PMID 19785086, PMID 19801505, PMID 19951979, PMID 19996412, PMID 20007560, PMID 20233970, PMID 20421452, PMID 20424435, PMID 20566896, PMID 20605573, PMID 20677256, PMID 20955402, PMID 20997138, PMID 21071497, PMID 21173101, PMID 21393329, PMID 21700776, PMID 21742281, PMID 21772051

NIH funding

U01HL065238 (8), 2014 (3), 2018 (3), 2019 (3), 2004 (2), 2016 (2), 2017 (2), R01DK057778 (2), U01HL065232 (2), U01HL065239 (2), U01HL065244 (2), U01HL065260 (2), 1OT2HL154815 (1), 2015 (1), 2023 (1)