Condition

Beta-Thalassemia clinical trials and research

• 11Clinical trials
• 1New in 30 days
• 26Countries

Newest Beta-Thalassemia clinical trials

All recruiting trials (11)

• Combining Thalidomide with Danazol and Prednisolone for difficult beta-thalassemia in children

  This trial tests whether adding danazol and prednisolone to thalidomide helps children with transfusion-dependent beta-thalassemia who did not respond to thalidomide alone.

  Recruiting Phase 3 Khyber Medical University Peshawar · Peshawar, Khyber Pakhtunkhwa · NCT07884773

• Partial stem cell transplant for sickle cell disease and beta-thalassemia using matched donors

  This study tests a partial stem cell transplant from matched donors to help people with severe sickle cell disease or beta-thalassemia see if more donor cells can persist long-term.

  Recruiting Phase 1/2 National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT07599176

• Testing 9MW3011 for patients with non-transfusion-dependent beta-thalassemia

  This study is testing a new drug called 9MW3011 to see if it is safe and effective for people with non-transfusion-dependent beta-thalassemia.

  Recruiting at 2 sites Phase 1 Mabwell (Shanghai) Bioscience Co., Ltd. · Nanning, Guangxi and 1 other locations · NCT06772766

• Gene editing of your own blood stem cells to boost fetal hemoglobin for severe sickle cell disease and beta-thalassemia

  This trial will test whether editing a specific gene in patients' own blood stem cells can raise fetal hemoglobin and reduce symptoms in people aged 13–40 with severe sickle cell disease or transfusion-dependent beta-thalassemia.

  Recruiting Phase 1 Boston Children's Hospital · Boston, Massachusetts · NCT06647979

• Long-term follow-up for patients treated with CS-101

  This study is checking how safe and effective the CS-101 treatment is for people who had it before, by following them for up to two years to see how they’re doing.

  Recruiting Children's Hospital of Fudan University · Shanghai, Shanghai Municipality · NCT06479616

• Observational study on patients with Beta-Thalassemia treated with betibeglogene autotemcel

  This study is looking at how well betibeglogene autotemcel works and its long-term safety for people with beta-thalassemia who have already received this treatment.

  Recruiting at 6 sites Genetix Biotherapeutics Inc. · Oakland, California and 5 other locations · NCT06271512

• Assessing the safety of luspatercept in Korean patients with myelodysplastic syndrome or beta thalassemia

  This study is testing how safe the drug luspatercept is for adult Korean patients with myelodysplastic syndrome or beta thalassemia while they are being treated.

  Recruiting Bristol-Myers Squibb · Seoul and 1 other locations · NCT06073860

• Evaluating a single dose of CTX001 for treating transfusion-dependent beta-thalassemia and severe sickle cell disease

  This study is testing a single dose of a new treatment called CTX001 to see if it can help people with transfusion-dependent beta-thalassemia or severe sickle cell disease feel better.

  Recruiting at 6 sites Phase 3 Vertex Pharmaceuticals Incorporated · New York, New York and 5 other locations · NCT05477563

• Evaluating the safety and pharmacokinetics of Luspatercept in children with beta-thalassemia

  This study is testing a new drug called Luspatercept to see if it's safe and how it works in children with beta-thalassemia, starting with teens and then including younger kids.

  Recruiting at 22 sites Phase 2 Celgene · Los Angeles, California and 25 other locations · NCT04143724

• Stem cell transplantation for blood disorders using specific cell depletion techniques

  This study is testing a new way of using stem cell transplants for children and young adults with blood disorders to see if removing certain cells helps them recover better and improve their quality of life.

  Recruiting Phase 2 New York Medical College · Valhalla, New York · NCT04099966

• Evaluating long-term safety of luspatercept for patients previously in other trials

  This study is testing the long-term safety of luspatercept for people with myelodysplastic syndromes, beta-thalassemia, and certain blood cancers who have already participated in other luspatercept trials.

  Recruiting at 27 sites Phase 3 Celgene · Los Angeles, California and 142 other locations · NCT04064060

Related topics

Quick answers

How many Beta-Thalassemia clinical trials are recruiting right now?

11, as of November 12, 2026. 1 of them was first registered in the last 30 days. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 26 countries. The most sites are in United States (7 trials), China (4 trials), Germany (3 trials), Italy (3 trials), Greece (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 3, Phase 2: 2, Phase 1: 2, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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