Clinical trials
Beta-Thalassemia clinical trials
Below are recruiting beta-thalassemia clinical trials, each written for real people, not researchers. We’re tracking 30 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 - 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 - 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 - 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 - NCT04099966RecruitingPhase 2
Stem cell transplant to treat high-risk blood cancers
This trial tests a specialized donor stem cell transplant to treat high-risk problems in the blood and bone marrow, including some cancers and non-cancers. It may help by using donor immune cells designed to better control the disease after transplant.
Valhalla, New YorkAges 1 day–30 years - 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 - 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+ - 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 - 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 - NCT06271512Recruiting
Follow-up study for beta-thalassemia patients treated with beti-cel
This study is for people with beta-thalassemia who have already received the gene therapy betibeglogene autotemcel (beti-cel). It collects information to learn how patients are doing after this treatment in real-world settings.
Oakland, CaliforniaAges Any age - 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 - NCT06364774RecruitingPhase 1/Phase 2
Gene therapy for transfusion-dependent beta thalassemia
This gene therapy trial uses a modified virus to deliver a healthy gene into your own stem cells. The goal is to help your body make normal red blood cells so you may need fewer or no transfusions.
Philadelphia, PennsylvaniaAges 18–40 - NCT06539169Recruiting
Following people with rare diseases over time
This study follows people with rare diseases over time to learn more about how these conditions progress and how they are treated. Joining may help researchers understand your disease better and find better ways to care for others.
Los Altos, CaliforniaAges Any age - 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 - NCT06772766RecruitingPhase 1
Study of 9MW3011 in adults with beta-thalassemia without regular transfusions
This trial tests a new drug, 9MW3011, in people with a type of beta-thalassemia that does not require regular blood transfusions. The goal is to see if the drug can safely help manage iron overload and improve hemoglobin levels.
Nanning, GuangxiAges 18–65 - NCT07055503Enrolling by invitation
Testing a new blood storage system for thalassemia transfusions
This study tests a special system (Hemanext One) that stores red blood cells with less oxygen, to see if it helps people with thalassemia who need regular transfusions. It may reduce side effects or improve how well the transfusions work.
Athens, AtticaAges 18 years+ - NCT06328764Enrolling by invitationEarly Phase 1
CS-101 for patients with beta-thalassemia
This trial tests a new treatment called CS-101 for people with beta-thalassemia who need regular blood transfusions. It aims to see if CS-101 can help reduce transfusion needs.
Nanning, GuangxiAges 6–35 - NCT04208529Enrolling by invitationPhase 3
Long-term follow-up after receiving CTX001 infusion
This study follows people for a long time after they received CTX001 in an earlier (parent) study. It helps researchers learn about long-term safety and outcomes of the treatment.
Palo Alto, CaliforniaAges 2 years+ - 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 - 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+ - 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 - NCT06363760Enrolling by invitation
Long-term follow-up after EDIT-301 gene therapy
This long-term follow-up study monitors people with sickle cell disease or transfusion-dependent beta-thalassemia who have already received the gene-editing therapy EDIT-301. It helps researchers understand how safe and effective the treatment is over time.
Oakland, CaliforniaAges 12–50
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Beta-Thalassemia trials by city
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Beta-Thalassemia trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for beta-thalassemia?
- Yes. Clin2 currently lists 30 recruiting beta-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 beta-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 beta-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.