Clinical trials
Thalassemia Majors (Beta-Thalassemia Major) clinical trials
Below are recruiting thalassemia majors (beta-thalassemia major) clinical trials, each written for real people, not researchers. We’re tracking 11 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07517133RecruitingPhase 3
Mitapivat for Children With Non-Transfusion-Dependent Thalassemia
This study tests a medicine called mitapivat in children ages 1–17 with thalassemia who don't need frequent blood transfusions. The medicine may help their bodies make more healthy red blood cells and reduce anemia symptoms.
Palo Alto, CaliforniaAges 1–17 - NCT07506863RecruitingPhase 3
Mitapivat for children with transfusion-dependent thalassemia
This study tests a study pill called mitapivat for children aged 1 to 17 with transfusion-dependent thalassemia. The goal is to see if mitapivat can reduce the need for blood transfusions and improve blood counts.
New York, New YorkAges 1–17 - 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 - NCT05773729Recruiting
Gene-modified own stem cell treatment for transfusion-dependent thalassemia
This trial tests a one-time treatment that uses your own (autologous) stem cells that have been changed by gene methods, then returned to you to help manage transfusion-dependent beta-thalassemia. It aims to check safety and whether it can reduce the need for frequent blood transfusions or improve blood health.
ShanghaiAges 3–18 - NCT06219239Recruiting
Gene therapy for transfusion-dependent beta-thalassemia
This trial tests a new gene therapy for people with beta-thalassemia who need regular blood transfusions. It uses a virus to deliver a working gene into your own stem cells, then those cells are put back into your body to help your body make healthy red blood cells.
Tianjin, Tianjin MunicipalityAges 3–35 - NCT04143724RecruitingPhase 2
Luspatercept for children with beta-thalassemia
This study tests a medicine called luspatercept to see if it safely helps children with beta-thalassemia need fewer blood transfusions or keep their hemoglobin levels stable. It is for children aged 6 to 17 who either get regular transfusions or have low hemoglobin and few transfusions.
Los Angeles, CaliforniaAges 6–17 - NCT06647979RecruitingPhase 1
Gene editing for severe sickle cell or thalassemia
This trial tests a new gene therapy that edits your own blood stem cells to help them make more fetal hemoglobin, which can prevent sickling and reduce the need for transfusions. It is for people with severe sickle cell disease or beta thalassemia who do not have a matched sibling donor.
Boston, MassachusettsAges 13–40 - 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 - NCT06568926Recruiting
Adherence to Oral Chelation in Beta Thalassemia
This study checks how well children with beta thalassemia stick to their daily oral chelation pill (deferasirox). It aims to find ways to help them take it more regularly, improving iron control and reducing complications.
SohagAges 2–18 - NCT06280378RecruitingPhase 1/Phase 2
KL003 stem cell treatment for beta-thalassemia
This trial tests a new treatment called KL003 for people with beta-thalassemia who need regular blood transfusions. The treatment uses your own stem cells (collected from your blood) to try to reduce or stop the need for transfusions.
Shanghai, Shanghai MunicipalityAges 3–35 - NCT07599176RecruitingPhase 1/Phase 2
Partial stem cell transplant for sickle cell disease from matched donors
This study tests a partial stem cell transplant from a matched donor to treat sickle cell disease. It may help reduce complications like strokes, organ damage, or pain crises.
Bethesda, MarylandAges 4–65
Hear when a new Thalassemia Majors (Beta-Thalassemia Major) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
Common questions
- Are there clinical trials for thalassemia majors (beta-thalassemia major)?
- Yes. Clin2 currently lists 11 recruiting thalassemia majors (beta-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 majors (beta-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 majors (beta-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.