Clinical trials
Thalassemia clinical trials
Below are recruiting thalassemia clinical trials, each written for real people, not researchers. We’re tracking 71 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - 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 - 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 - NCT05477563RecruitingPhase 3
One-time gene therapy for severe sickle cell and transfusion anemia
This trial tests a single dose of gene therapy (CTX001) to improve outcomes for people with severe sickle cell disease or transfusion-dependent beta-thalassemia. It aims to see how well it works and whether it is safe, especially after a planned stem cell transplant process.
New York, New YorkAges 12–35 - NCT05736419RecruitingPhase 2
Immune-suppressing treatment before stem cell transplant for blood disorders
This Phase 2 study tests an immune-suppressing treatment for people with sickle cell disease or beta-thalassemia who are preparing for a stem cell transplant from a donor. It aims to help the transplant work better by changing the immune response.
Basking Ridge, New JerseyAges 2–50 - NCT07215975Recruiting
Luspatercept for adults with transfusion-dependent beta-thalassemia
This study looks at how well a medication called Luspatercept works for adults with transfusion-dependent beta-thalassemia, a blood condition that requires regular blood transfusions. Researchers want to understand how it helps people in the Middle East.
Al JahraAges 18 years+ - NCT07673302Recruiting
Study of drugs to boost fetal hemoglobin in beta thalassemia
This trial tests if certain drugs can safely increase fetal hemoglobin (a type of hemoglobin that helps carry oxygen) in people with beta thalassemia major. It may offer a new way to manage the condition and reduce the need for transfusions.
Rawalpindi, Punjab ProvinceAges Any age - NCT06657391RecruitingPhase 2
Study of a new drug to prevent graft-versus-host disease in thalassemia patients
This trial tests a drug called an anti-CD25 antibody to see if it can prevent graft-versus-host disease (a condition where donor cells attack the body) in people with transfusion-dependent thalassemia who are getting a stem cell transplant from a partially matched or unrelated donor.
Maoming, GuangdongAges 3–20 - NCT05991336Recruiting
Growth and development after gene therapy for beta-thalassemia in children
This study looks at how children with transfusion-dependent beta-thalassemia grow and develop after gene therapy. It compares children who have had gene therapy with those who have not, and also with healthy children of the same age and gender.
Tianjin, Tianjin MunicipalityAges 3–14 - NCT05864170RecruitingEarly Phase 1
Tested injection for severe transfusion-dependent thalassemia
This early-stage study tests the safety and possible benefits of an injection called HGI-001 for people with severe transfusion-dependent beta thalassemia. It may help by preparing you for stem cell transplant, but you must meet several health and lab criteria and be able to return often for follow-up tests.
Shenzhen, GuangdongAges 18–35 - NCT05851105RecruitingEarly Phase 1
Study drug for severe alpha thalassemia in people needing regular transfusions
This early-phase trial studies HGI-002 to see if it’s safe and can help people with severe alpha thalassemia who need frequent blood transfusions. It also requires planning for a special treatment approach that includes busulfan pre-treatment and stem cell transplantation.
Nanning, GuangxiAges 12–35 - 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 - 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 - 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 - 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 - 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 - NCT05757245RecruitingPhase 1
Safety and effectiveness of GMCN-508A for transfusion-dependent alpha thalassemia
This Phase 1 study tests a new medicine (GMCN-508A) to see how safe it is and whether it can help people with transfusion-dependent alpha thalassemia. Participants are also expected to follow a treatment plan that includes stem cell transplantation preparation.
Nanning, GuangxiAges 5–35 - NCT07680803RecruitingPhase 2
Gene therapy for transfusion-dependent beta-thalassemia
This trial tests a one-time gene therapy using your own stem cells to help you produce healthy red blood cells and reduce or eliminate the need for regular blood transfusions. It is for people with transfusion-dependent beta-thalassemia who are between 3 and 35 years old.
Rome, LazioAges 3–35 - NCT06302491RecruitingPhase 2
Testing AND017 for beta-thalassemia
This trial tests an oral medication called AND017 for people with beta-thalassemia. It aims to see if the drug can safely help reduce the need for blood transfusions or improve hemoglobin levels.
Guangzhou, GuangdongAges 18–65 - NCT05426252RecruitingPhase 1/Phase 2
Lower-toxicity bone marrow conditioning for high-risk thalassemia
This trial tests a modified, lower-toxicity preparation (conditioning) before a bone marrow transplant in children and teens with high-risk thalassemia. It aims to reduce treatment side effects while still allowing transplant success for people who need a donor.
Toronto, OntarioAges 1–18 - NCT05508932Recruiting
Heart rhythm study for people with transfusion-dependent beta-thalassemia
This trial studies atrial fibrillation (an irregular heartbeat) in people with beta-thalassemia who need regular blood transfusions. It may help doctors better understand how this heart rhythm problem affects you and guide future care.
FerraraAges 18 years+ - 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+ - NCT05762510RecruitingEarly Phase 1
Testing a gene-based treatment for transfusion-dependent thalassemia
This early study tests the safety and potential benefit of the LentiRed gene-based drug in people with transfusion-dependent beta-thalassemia (TDT) who are planning an autologous stem cell transplant. It may help reduce the severity of anemia, but the main goal at this stage is to check safety and side effects.
Nanning, GuangxiAges 5–35
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Thalassemia trials by city
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Thalassemia trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for thalassemia?
- Yes. Clin2 currently lists 71 recruiting thalassemia 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 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 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.