Clinical trials
Transfusion Dependent Thalassemia clinical trials
Below are recruiting transfusion dependent thalassemia clinical trials, each written for real people, not researchers. We’re tracking 25 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 - 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 - 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 - 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 - 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+ - 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 - 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 - 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 - 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 - 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 - 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 - NCT07207577Recruiting
Exercise and fatigue in beta-thalassemia
This study looks at why people with transfusion-dependent beta-thalassemia may feel tired or have trouble exercising. It compares patients with healthy volunteers matched by age and gender to understand the factors involved.
ChambéryAges 18 years+ - 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 - NCT06664541Enrolling by invitation
Gene therapy decisions and quality of life
This study looks at how people with blood diseases and their families make decisions about gene therapy, and how it affects their quality of life. It involves interviews or surveys to understand your experience.
Washington D.C., District of ColumbiaAges 18 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 - 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+ - 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+ - NCT07338344RecruitingPhase 2
Luspatercept with thalidomide for beta-thalassemia patients
This study tests whether a combination of two drugs, luspatercept and thalidomide, can help people with beta-thalassemia who need regular blood transfusions. It aims to reduce the number of transfusions needed.
Shenzhen, GuangdongAges 18–75
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Common questions
- Are there clinical trials for transfusion dependent thalassemia?
- Yes. Clin2 currently lists 25 recruiting transfusion dependent 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 transfusion dependent 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 transfusion dependent 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.