Clinical trials
Thalassemia Major clinical trials
Below are recruiting thalassemia major clinical trials, each written for real people, not researchers. We’re tracking 12 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06734520Recruiting
Super transplant for severe beta-thalassemia
This trial tests a special type of stem cell transplant (called a super transplant) for children with a severe form of beta-thalassemia. It may be an option if you cannot or choose not to have a standard stem cell transplant or gene therapy.
Guangzhou, GuangdongAges 7–10 - NCT04872179Recruiting
Registry for people affected by alpha thalassemia
This is a research registry collecting information about people with alpha thalassemia to support ongoing studies. It may help the team better understand how fetal stem cell treatments are working and for whom they may be most suitable.
San Francisco, CaliforniaAges Any age - NCT07112703Recruiting
Breathing exercise study for children with thalassemia
This study tests if a breathing exercise device (incentive spirometer) can improve lung function and daily activity in children with thalassemia who receive regular blood transfusions. It aims to help them breathe easier and be more active.
ZagazigAges 6–10 - NCT06465550RecruitingPhase 1
Gene therapy for beta-thalassemia in children and teens
This trial tests a new gene therapy (BD211) for children and teens with severe beta-thalassemia who need regular blood transfusions. The treatment uses your own blood stem cells, modified with a working gene, to help your body make healthy red blood cells and reduce the need for transfusions.
Guangzhou, GuandongAges 3–35 - NCT06041620Recruiting
Gene-edited stem cell therapy for beta-thalassemia
This trial tests a new treatment that uses your own blood stem cells, edited with CRISPR technology, to reduce or eliminate the need for blood transfusions in people with beta-thalassemia.
Tianjin, Tianjin MunicipalityAges 3–35 - NCT05745532RecruitingEarly Phase 1
Autologous stem cell treatment to improve beta thalassemia major
This early-phase trial tests a new way to restore working blood-making cells using your own stem cells for people with transfusion-dependent beta thalassemia major. The goal is to improve safety and blood results, and possibly reduce problems from the disease.
Shenzhen, GuangdongAges 8–16 - NCT05776173Recruiting
Gene-modified stem cell treatment for transfusion-dependent thalassemia
This trial tests whether giving you your own (autologous) gene-modified blood stem cells can improve transfusion-dependent beta-thalassemia and its safety. It may help people who do not have a fully matched donor and need an alternative approach to care.
ShanghaiAges 6–35 - NCT06490627RecruitingPhase 2
thalidomide for transfusion-dependent beta thalassemia
This trial tests different doses of thalidomide to see if it can reduce or eliminate the need for blood transfusions in people with beta thalassemia major or intermedia.
Karachi, SindhAges 8–35 - NCT05693909RecruitingPhase 2
SP-420 for iron overload in certain blood disorders
This Phase 2 study tests SP-420 to help people with transfusion-related iron overload from either beta-thalassemia or low-risk myelodysplastic syndromes. It aims to see if the treatment can improve how the body handles excess iron, which builds up after repeated transfusions.
CopenhagenAges 18 years+ - NCT03653338RecruitingPhase 1/Phase 2
Bone marrow transplant using donor blood to treat sickle cell
This trial tests a specific type of bone marrow transplant for people with sickle cell disease (or certain other blood disorders) who have had serious complications despite standard care. It aims to see if removing the patient’s immune cells and using a matched donor’s cells can improve outcomes and reduce complications.
Pittsburgh, PennsylvaniaAges 5–40 - NCT06743477Recruiting
Super transplant for severe thalassemia in children
This trial tests a new type of transplant (called 'super transplantation') for children with severe thalassemia. It aims to help those who cannot get a fully matched donor or have chosen not to have standard treatments.
Kunming, YunnanAges 7–12 - NCT06466304Recruiting
Breathing muscle training for children with beta thalassemia major
This study tests whether special breathing exercises can help children with beta thalassemia major. It aims to strengthen the muscles used for breathing, which may improve overall health.
GizaAges 8–12
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Common questions
- Are there clinical trials for thalassemia major?
- Yes. Clin2 currently lists 12 recruiting thalassemia major studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a thalassemia major trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a thalassemia major trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.