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
- NCT05799118Recruiting
Genetic modifiers study for inherited blood disorders
This study looks at how your genes can affect inherited blood conditions like sickle cell disease and thalassemia. It may help researchers understand why symptoms differ from person to person, which could guide better future treatments.
Boston, MassachusettsAges 2 years+ - NCT06041620Recruiting
Gene-edited stem cell therapy for beta-thalassemia
This trial tests a new treatment that uses your own blood stem cells, edited with CRISPR technology, to reduce or eliminate the need for blood transfusions in people with beta-thalassemia.
Tianjin, Tianjin MunicipalityAges 3–35 - NCT06073860Recruiting
Safety study of Luspatercept for MDS or thalassemia in Korea
This study is checking the safety of a drug called Luspatercept (brand name Reblozyl) for people in Korea who have myelodysplastic syndrome (a blood disorder) or beta-thalassemia (a type of inherited anemia). It is designed to monitor how safe the drug is when used as prescribed by your doctor.
SeoulAges 19 years+ - 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 - 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 - 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 - NCT06591936Recruiting
Genetic study of alpha thalassemia in children
This trial looks at the genetic profiles of children with suspected alpha thalassemia, a type of inherited anemia. It aims to better understand this condition in children treated at Sohag University Hospital.
SohagAges birth–18 years - 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 - 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+ - 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 - 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 - 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+ - 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 - 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+ - 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 - 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+ - NCT05664737RecruitingPhase 2
Luspatercept for adults with alpha thalassemia
This study tests a medicine called luspatercept to see if it can help people with alpha thalassemia need fewer blood transfusions. It is for adults and some teens with this condition.
Halifax, Nova ScotiaAges 12 years+ - 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 - 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 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.