Thalassemia Major Clinical Trials

10 recruitingLast updated: May 13, 2026

There are 10 actively recruiting thalassemia major clinical trials across 5 countries. Studies span Not Applicable, Phase 2, Phase 1. Top locations include Tianjin, Tianjin Municipality, China, Giza, Egypt, Guangzhou, Guangdong, China. Updated daily from ClinicalTrials.gov.


Thalassemia Major Trials at a Glance

10 actively recruiting trials for thalassemia major are listed on ClinicalTrialsFinder across 6 cities in 5 countries. The largest study group is Not Applicable with 6 trials, with the heaviest enrollment activity in Tianjin, Giza, and Guangzhou. Lead sponsors running thalassemia major studies include Institute of Hematology & Blood Diseases Hospital, China, Guangzhou Women and Children's Medical Center, and Ahram Canadian University.

Browse thalassemia major trials by phase

Treatments under study

About Thalassemia Major Clinical Trials

Looking for clinical trials for Thalassemia Major? There are currently 5 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 Thalassemia Major 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 Thalassemia Major 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 110 of 10 trials

Recruiting
Not Applicable

Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia

Thalassemia MajorThalassemia IntermediaGrowth Delay+1 more
Institute of Hematology & Blood Diseases Hospital, China369 enrolled1 locationNCT06931912
Recruiting
Phase 2

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes

Myelodysplastic SyndromeBeta Thalassemia Major Anemia
Pharmacosmos A/S90 enrolled1 locationNCT05693909
Recruiting
Phase 1Phase 2

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Diamond-Blackfan AnemiaSickle Cell Anemiabeta thalassemia major
Paul Szabolcs5 enrolled1 locationNCT03653338
Recruiting
Not Applicable

Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major

Pulmonary FunctionChild, OnlyThalassemia Major+2 more
Rawan Mohammed Khairy Mostafa Elsawy50 enrolled1 locationNCT07112703
Recruiting
Phase 2

Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia

Genetic DiseaseFetal HemoglobinThalassemia Major
National Institute of Blood and Marrow Transplant (NIBMT), Pakistan54 enrolled1 locationNCT06490627
Recruiting

International Registry of Patients With Alpha Thalassemia

Alpha-ThalassemiaAlpha Thalassemia MajorAlpha Thalassemia Minor
University of California, San Francisco500 enrolled1 locationNCT04872179
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
Not Applicable

Inspiratory Muscle Training in Children With Beta Thalasemia

beta thalassemia major
Ahram Canadian University40 enrolled1 locationNCT06466304
Recruiting
Not Applicable

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia

Thalassemia Major
Shanghai BDgene Co., Ltd.10 enrolled1 locationNCT05776173
Recruiting
Not Applicable

Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

Thalassemia MajorThalassemia (beta)
Institute of Hematology & Blood Diseases Hospital, China2 enrolled1 locationNCT06041620