beta-Thalassemia: Clinical Trials

124 completed, 54 unknown, 29 recruiting, 18 active not recruiting. 87 Phase 2, 51 Not applicable, 35 Phase 1, 32 Phase 3, 17 Phase 4.

By phase and status

Phase	Trials	Status
Phase 2	87	Completed 42, Unknown 13, Active not recruiting 9, Recruiting 9, Terminated 7, Withdrawn 4, Enrolling by invitation 2, Not yet recruiting 1
Not applicable	51	Completed 25, Unknown 14, Not yet recruiting 4, Recruiting 4, Active not recruiting 2, Suspended 1, Terminated 1
Phase 1	35	Completed 14, Active not recruiting 6, Recruiting 6, Terminated 4, Unknown 3, Enrolling by invitation 1, Withdrawn 1
Phase 3	32	Completed 16, Unknown 9, Recruiting 3, Active not recruiting 1, Enrolling by invitation 1, Not yet recruiting 1, Terminated 1
Phase 4	17	Completed 11, Unknown 5, Active not recruiting 1
Early Phase 1	5	Completed 2, Enrolling by invitation 1, Recruiting 1, Withdrawn 1

Follow beta-Thalassemia: new trials by email Literature Review of beta-Thalassemia trials Research Report on beta-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
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
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
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
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	Enrolling by invitation	Fulcrum Therapeutics	50	2026-03-30	2026-04-14
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
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452 - Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)	Phase 1	Recruiting	Daniel Bauer	10	2025-12-01	2026-02-03
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
The Effect of Laughter Yoga in Beta Thalassaemia Major Patients: a Randomised Controlled Trial laughter yoga - Beta Thalassemia Major, Thalassemia Majors (Beta-Thalassemia Major)	Not applicable	Not yet recruiting	Ataturk University	60	2025-06-01	2025-05-20
A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection Beta-Thalassemia		Enrolling by invitation	CorrectSequence Therapeutics Co., Ltd	8	2025-05-15	2026-02-10
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia ALS20 - Beta-Thalassemia	Phase 1 / Phase 2	Recruiting	Children's Hospital of Philadelphia	12	2025-04-14	2026-09-23
Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major Incentive spirometer exercises, Diaphragmatic breathing, Pursed lip breathing: - Incentive Spirometer, Pulmonary Function	Not applicable	Recruiting	Rawan Mohammed Khairy Mostafa Elsawy	50	2025-04-07	2025-08-08
A Long-term Follow-up Study in Participants Who Received CS-101 Safety and efficacy assessments - Beta-Thalassemia		Enrolling by invitation	CorrectSequence Therapeutics Co., Ltd	5	2025-01-18	2026-02-10

Showing 20 of 264. Sign in to see all of them.

Narrower conditions

Anemia, Sickle Cell (58)  ·  Thalassemia (43)  ·  Iron Overload (30)  ·  Hematologic Diseases (19)  ·  Hemoglobinopathies (18)  ·  Anemia (12)  ·  Genetic Diseases, Inborn (12)  ·  Anemia, Diamond-Blackfan (10)  ·  Myelodysplastic Syndromes (9)  ·  alpha-Thalassemia (8)  ·  Anemia, Aplastic (6)  ·  Rare Diseases (6)  ·  Diabetes Mellitus (5)  ·  Osteoporosis (5)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (264)  ·  Hemic and Lymphatic Diseases (264)  ·  Anemia, Hemolytic (262)  ·  Anemia, Hemolytic, Congenital (262)  ·  Anemia (252)  ·  Genetic Diseases, Inborn (252)  ·  Hemoglobinopathies (246)  ·  Hematologic Diseases (245)  ·  Thalassemia (221)  ·  Nutritional and Metabolic Diseases (51)  ·  Metabolic Diseases (50)  ·  Iron Metabolism Disorders (30)  ·  Pathological Conditions, Signs and Symptoms (27)  ·  Bone Marrow Diseases (21)  ·  Pathologic Processes (21)

Interventions

Deferasirox (17)  ·  Deferoxamine (12)  ·  Hydroxyurea (12)  ·  luspatercept (12)  ·  Deferiprone (11)  ·  Busulfan (9)  ·  fludarabine (9)  ·  Alemtuzumab (8)  ·  Cyclophosphamide (8)  ·  Mycophenolic Acid (7)  ·  Tacrolimus (7)  ·  Thalidomide (6)  ·  fludarabine phosphate (6)  ·  Isoflurophate (5)  ·  thymoglobulin (5)

Sponsors

Assiut University (15)  ·  Ain Shams University (10)  ·  Tanta University (9)  ·  Celgene (8)  ·  Novartis Pharmaceuticals (8)  ·  CorrectSequence Therapeutics Co., Ltd (5)  ·  Institute of Hematology & Blood Diseases Hospital, China (5)  ·  Nanfang Hospital, Southern Medical University (5)  ·  National Heart, Lung, and Blood Institute (NHLBI) (5)  ·  Bioray Laboratories (4)  ·  Bristol-Myers Squibb (4)  ·  First Affiliated Hospital of Guangxi Medical University (4)  ·  Genetix Biotherapeutics Inc. (4)  ·  Vertex Pharmaceuticals Incorporated (4)  ·  Boston University (3)

Countries

United States (86)  ·  Italy (44)  ·  China (37)  ·  Egypt (32)  ·  Greece (22)  ·  Thailand (21)  ·  Canada (20)  ·  Lebanon (19)  ·  Turkey (Türkiye) (18)  ·  United Kingdom (16)  ·  Germany (10)  ·  Pakistan (10)  ·  France (7)  ·  Israel (7)  ·  Spain (6)

Top investigators

John F Tisdale, M.D. (3)  ·  Paul Szabolcs, MD (3)  ·  Yongrong Lai, M.D. (3)  ·  Himal Lal Thakar, MD (2)  ·  Houman Hemmati, MD (2)  ·  Jun Shi, PhD (2)  ·  Mohsen S. Elalfy, professour (2)  ·  Patricia J. Giardina, MD (2)  ·  Petros Kountouris, PhD (2)  ·  Rongmei Liang, PhD (2)  ·  Xiaowen Zhai, M.D. (2)

Results publications

PMID 37975597, PMID 30617198, PMID 33570654, PMID 34175041, PMID 36240296, PMID 37704579, PMID 40862696, PMID 41857682, PMID 42233421, PMID 42252696, PMID 42705233, PMID 14550808, PMID 16273116, PMID 16737942, PMID 17951527, PMID 18298856, PMID 18568278, PMID 19236376, PMID 19724055, PMID 19785086, PMID 20007560, PMID 20233970, PMID 20421452, PMID 20424435, PMID 20955402, PMID 20997138, PMID 21173101, PMID 21393329, PMID 21700776, PMID 21742281, PMID 22251482, PMID 22571408, PMID 23553596, PMID 23927461, PMID 24081970, PMID 24452753, PMID 24797180, PMID 25058217, PMID 25212456, PMID 25222041, PMID 25600572, PMID 26348869, PMID 26653680, PMID 27071063, PMID 27326168, PMID 27862048, PMID 28249145, PMID 29119631, PMID 29155313, PMID 29669226

NIH funding

U01HL065238 (3), 2014 (2), 2018 (2), 2019 (2), U01HL065232 (2), U01HL065239 (2), U01HL065244 (2), U01HL065260 (2), 1OT2HL154815 (1), 2015 (1), 2016 (1), 2017 (1), 2023 (1), P01HL053750 (1), R01DK116126 (1)