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
- NCT07725263Recruiting
Low-dose antifungal for kids with low white blood cells
This trial tests a low dose of an antifungal medicine (liposomal amphotericin B) to prevent serious fungal infections in children whose immune system is weak after cancer treatment or a stem cell transplant. It's for kids aged 3 to 17 who are at high risk for these infections.
Haikou, HainanAges 3–17 - 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 - 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 - 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+ - 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 - 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 - 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 - 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 - NCT05904093RecruitingPhase 1
Check if increasing fostamatinib doses are safe in sickle cell
This Phase 1 study tests whether increasing doses of fostamatinib are safe and tolerated in adults with stable sickle cell disease. It may help researchers understand if the drug could be a future treatment option for people with stable sickle cell.
Bethesda, MarylandAges 18–65 - NCT06107400RecruitingEarly Phase 1
RM-004 cell therapy for Hemoglobin H-Constant Spring disease
This early-phase trial tests a new cell therapy called RM-004 for people with Hemoglobin H-Constant Spring disease, a form of alpha thalassemia that requires regular blood transfusions. The goal is to see if the treatment is safe and can reduce or eliminate the need for transfusions.
Nanning, GuangxiAges 12–35 - NCT06213402Recruiting
European registry for rare anemia disorders
This study is creating a European registry (database) to collect information about people with rare anemia disorders. By joining, you help researchers better understand these conditions and improve care for yourself and others.
Barcelona, CataloniaAges birth–100 years - NCT06250595Recruiting
European rare blood disorder registry
This study is building a registry of people with rare blood diseases. By joining, you help doctors learn more about these conditions and improve future care.
Barcelona, CataloniaAges Up to 100 years - 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 - NCT06314529Recruiting
Long-term follow-up of BRL-101 for thalassemia
This study follows people who have already received BRL-101 treatment for transfusion-dependent beta-thalassemia to check their health over time. It helps doctors understand how well the treatment works in the long run.
Nanning, GuangxiAges 5–35 - NCT06466304Recruiting
Breathing muscle training for children with beta thalassemia major
This study tests whether special breathing exercises can help children with beta thalassemia major. It aims to strengthen the muscles used for breathing, which may improve overall health.
GizaAges 8–12 - 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 - 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 - 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 - NCT06655662RecruitingPhase 1
Gene therapy for severe beta-thalassemia
This trial tests a one-time gene therapy using your own blood stem cells to treat severe beta-thalassemia. It may help you stop needing regular blood transfusions.
GuangxiAges 6–35 - NCT07177300RecruitingPhase 4
Hydroxyurea for sickle cell disease in children
This trial tests different ways to start hydroxyurea for children with sickle cell anemia. The goal is to find the safest and most effective approach to help manage symptoms and prevent complications.
Cincinnati, OhioAges 6 months+ - 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 - 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+
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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.