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
- 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+ - NCT07112703Recruiting
Breathing exercise study for children with thalassemia
This study tests if a breathing exercise device (incentive spirometer) can improve lung function and daily activity in children with thalassemia who receive regular blood transfusions. It aims to help them breathe easier and be more active.
ZagazigAges 6–10 - 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+ - 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 - 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 - 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 - 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 - 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 - 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+ - 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 - 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+ - NCT04432623Enrolling by invitationPhase 1/Phase 2
Test drug for adults with beta thalassemia intermedia
This trial studies an investigational medicine in adults with beta thalassemia intermedia (including non-transfusion dependent thalassemia). The goal is to see how well the treatment works and how safe it is for people whose hemoglobin runs in a certain range.
Oakland, CaliforniaAges 18 years+ - 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+ - 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 - 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 - 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 - 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 - 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 - 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 - 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 - 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 - 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+ - 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+
Hear when a new Thalassemia trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
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.