Clinical trials
Thalassemia clinical trials
Below are recruiting thalassemia clinical trials, each written for real people, not researchers. We’re tracking 64 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - NCT06717932Enrolling by invitation
Long-term safety study of CS-101 injection
This study follows up with people who already received the CS-101 injection in a previous trial to check its safety and how well it works over time.
NanningAges 12–35 - 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 - NCT06685536Enrolling by invitation
Long-term follow-up for CS-101 recipients
This study follows up on people who have already received the CS-101 infusion in a previous trial. It aims to check how participants are doing over time. There are no restrictions to join if you already received CS-101.
Nanning, GuangxiAges 6–35 - 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+ - 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 - NCT06609226RecruitingPhase 3
Long-term safety study of etavopivat for sickle cell disease or thalassaemia
This study looks at the long-term safety and benefits of taking etavopivat for people with sickle cell disease or thalassaemia who have already been helped by it in a previous trial. It aims to see if staying on the treatment continues to be safe and effective.
Birmingham, AlabamaAges 2 years+ - NCT06479616Recruiting
Long-term follow-up for CS-101 recipients
This study checks on participants who previously received the experimental treatment CS-101. It aims to track their health over time to see how safe and effective the treatment is in the long run.
Shanghai, Shanghai MunicipalityAges 3–17 - NCT06313398RecruitingEarly Phase 1
Measuring red blood cell lifespan in sickle cell disease
This study uses a harmless vitamin (biotin) to label your red blood cells and track how long they survive. It aims to understand how different blood disorders affect red blood cell lifespan, which may help improve treatments.
Bethesda, MarylandAges 18–100 - NCT06298630Recruiting
Long-term study of BRL-101 for transfusion-dependent beta-thalassemia
This study follows patients who have already been treated with BRL-101 for transfusion-dependent beta-thalassemia. Its goal is to track long-term outcomes and safety.
Guangzhou, GuangdongAges 3–35 - NCT05777993Enrolling by invitationPhase 4
Continued access to mitapivat for prior study participants
This study provides continued access to the drug mitapivat for people who have already been in a mitapativ study and are benefiting from it.
Anderlecht, Brussels CapitalAges 18 years+ - NCT05414045Recruiting
Transplanting your own frozen testicle tissue for future fertility
This trial studies transplanting frozen testicular tissue back into the body to help restore fertility later. It is meant for people who preserved testicular tissue as children before cancer or a blood disorder treatment.
BrusselsAges 18–50 - NCT04398628Recruiting
Study of non-cancer blood disorders in people with bleeding or clotting
This study follows people with inherited or acquired non-cancer blood disorders to better understand symptoms, causes, and how different treatments work over time. You may be placed into a disorder-specific group, depending on your diagnosis and testing results.
Phoenix, ArizonaAges Any age - NCT04064060RecruitingPhase 3
Long-term safety follow-up for luspatercept study participants
This trial studies the long-term safety of luspatercept (ACE-536) in people who already took part in an earlier luspatercept study. It mainly helps doctors better understand ongoing side effects and overall safety over time.
Los Angeles, CaliforniaAges 18 years+ - NCT03937817Recruiting
Donate samples to study globin gene variants
This study collects common body samples to learn how different globin gene variants work and how they relate to health. Your samples may also be used later for new research, including genetic testing.
Bethesda, MarylandAges 18–70 - 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
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Common questions
- Are there clinical trials for thalassemia?
- Yes. Clin2 currently lists 64 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.