Beta-Thalassemia Clinical Trials

21 recruitingLast updated: June 18, 2026

There are 21 actively recruiting beta-thalassemia clinical trials across 27 countries. Studies span Phase 2, Phase 1, Not Applicable, Phase 3, Early Phase 1. Top locations include Tianjin, Tianjin Municipality, China, New York, New York, United States, Shanghai, Shanghai Municipality, China. Updated daily from ClinicalTrials.gov.


Beta-Thalassemia Trials at a Glance

21 actively recruiting trials for beta-thalassemia are listed on ClinicalTrialsFinder across 6 cities in 27 countries. The largest study group is Phase 2 with 7 trials, with the heaviest enrollment activity in Tianjin, New York, and Shanghai. Lead sponsors running beta-thalassemia studies include Celgene, Institute of Hematology & Blood Diseases Hospital, China, and Children's Hospital of Fudan University.

Browse beta-thalassemia trials by phase

Treatments under study

About Beta-Thalassemia Clinical Trials

Looking for clinical trials for Beta-Thalassemia? There are currently 15 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Beta-Thalassemia trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Beta-Thalassemia clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 21 trials

Recruiting
Phase 1Phase 2

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

Beta-ThalassemiaSickle Cell Disease
National Heart, Lung, and Blood Institute (NHLBI)90 enrolled1 locationNCT07599176
Recruiting
Phase 3

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-ThalassemiaMyelodysplastic Syndromes (MDS)Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis
Celgene665 enrolled143 locationsNCT04064060
Recruiting
Phase 1Phase 2

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia
Children's Hospital of Philadelphia12 enrolled1 locationNCT06364774
Recruiting
Phase 3

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Beta-ThalassemiaSickle Cell DiseaseHematologic Diseases+4 more
Vertex Pharmaceuticals Incorporated26 enrolled6 locationsNCT05477563
Recruiting
Phase 2

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Regeneron Pharmaceuticals95 enrolled26 locationsNCT06421636
Recruiting
Phase 1

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

Beta-ThalassemiaSickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)+1 more
Daniel Bauer10 enrolled1 locationNCT06647979
Recruiting
Phase 2

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Beta-Thalassemia
Celgene99 enrolled26 locationsNCT04143724
Recruiting
Phase 1Phase 2

An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia

Beta-Thalassemia
Lantu Biopharma6 enrolled2 locationsNCT06308159
Recruiting
Not Applicable

A Long-term Follow-up Study in Participants Who Received CS-101

Beta-Thalassemia
Children's Hospital of Fudan University5 enrolled1 locationNCT06479616
Recruiting
Phase 2

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Hodgkin LymphomaNon-Hodgkin LymphomaAcute Leukemia+6 more
Mitchell Cairo20 enrolled1 locationNCT04099966
Recruiting
Not Applicable

Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients

Transfusion Dependent Beta-Thalassemia
Institute of Hematology & Blood Diseases Hospital, China3 enrolled1 locationNCT06219239
Recruiting
Phase 1Phase 2

Β-Thalassemia Treatment with KL003 Cell Injection

Transfusion Dependent Beta-Thalassemia
Kanglin Biotechnology (Hangzhou) Co., Ltd.41 enrolled2 locationsNCT06280378
Recruiting

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

Beta-Thalassemia
Genetix Biotherapeutics Inc.150 enrolled6 locationsNCT06271512
Recruiting
Phase 1

A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia

Beta-Thalassemia
Mabwell (Shanghai) Bioscience Co., Ltd.40 enrolled2 locationsNCT06772766
Recruiting
Not Applicable

Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia

Thalassemia Majors (Beta-Thalassemia Major)Haplo-identical Donors
Guangzhou Women and Children's Medical Center3 enrolled2 locationsNCT06734520
Recruiting

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Myasthenia GravisAmyloidosisAlpha-Thalassemia+14 more
xCures1,000 enrolled1 locationNCT06539169
Recruiting

A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia

Myelodysplastic SyndromeBeta-Thalassemia
Bristol-Myers Squibb104 enrolled2 locationsNCT06073860
Recruiting
Not Applicable

Adherence of Beta Thalssemia Patients to Oral Chelation Therapy

Beta-Thalassemia
Sohag University200 enrolled1 locationNCT06568926
Recruiting

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

CancerLymphomaLeukemia+44 more
Sanguine Biosciences20,000 enrolled1 locationNCT05635266
Recruiting

Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Gene TherapyTransfusion Dependent Beta-Thalassemia
Institute of Hematology & Blood Diseases Hospital, China100 enrolled1 locationNCT05991336