Condition

Thalassemia clinical trials and research

• 18Clinical trials
• 9NIH research projects
• 1New in 30 days
• 21Countries

Newest Thalassemia clinical trials

All recruiting trials (18)

• 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

• Low-dose liposomal amphotericin B to prevent invasive fungal infections in neutropenic children

  This study tests whether giving a fixed low dose of liposomal amphotericin B can prevent invasive fungal infections in children aged 3–17 who are severely neutropenic after hematopoietic stem cell transplantation or similar high-risk conditions.

  Recruiting Haikou Affiliated Hospital of Central South University Xiangya School of Medicine · Haikou, Hainan · NCT07725263

• Safety and Effect of HbF Inducers in Beta Thalassemia

  This study tests whether a combination of thalidomide and hydroxyurea can safely raise fetal hemoglobin and reduce transfusions in people with beta thalassemia.

  Recruiting Riphah International University · Rawalpindi, Punjab Province · NCT07673302

• Luspatercept for adults with transfusion-dependent beta-thalassemia in the Middle East

  This project tests whether luspatercept can reduce blood transfusion needs in adults with transfusion-dependent beta-thalassemia in the Middle East.

  Recruiting at 4 sites Bristol-Myers Squibb · Al Jahra and 12 other locations · NCT07215975

• Gene therapy for patients with β-thalassemia using modified stem cells

  This study is testing a new gene therapy using modified stem cells to see if it can help young people with β-thalassemia reduce their need for blood transfusions.

  Recruiting at 3 sites Phase 1 Shenzhen Hemogen · Guangxi and 2 other locations · NCT06655662

• Long-term treatment with Etavopivat for sickle cell disease and thalassemia

  This study is testing how safe and effective a new medication called Etavopivat is for people with sickle cell disease and thalassemia who have already seen benefits from it.

  Recruiting at 45 sites Phase 3 Novo Nordisk A/S · Birmingham, Alabama and 106 other locations · NCT06609226

• Gene therapy for treating β-thalassemia Major

  This study is testing a new gene therapy to see if it can safely help young people with severe β-thalassemia reduce their need for blood transfusions.

  Recruiting at 3 sites Phase 1 Shanghai BDgene Co., Ltd. · Guangzhou, Guandong and 2 other locations · NCT06465550

• Understanding red blood cell lifespan in sickle cell disease and hemoglobin disorders

  This study is trying to see how long red blood cells last in people with sickle cell disease and other blood disorders to help improve their treatment.

  Recruiting Early Phase 1 National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT06313398

• Treatment of β-thalassemia with AND017

  This study is testing a new drug called AND017 to see if it can help people with β-thalassemia feel better compared to standard treatments.

  Recruiting at 4 sites Phase 2 Kind Pharmaceuticals LLC · Guangzhou, Guangdong and 4 other locations · NCT06302491

• Platform for Rare Blood Disorders in Europe

  This study is trying to gather and share information about rare blood disorders across Europe to help researchers understand these conditions better and improve treatment options for patients.

  Recruiting Hospital Universitari Vall d'Hebron Research Institute · Barcelona, Catalonia · NCT06250595

• European platform for collecting data on rare anemia disorders

  This study is trying to gather important information from people with rare anemia disorders across Europe to help improve their treatment and care.

  Recruiting Hospital Universitari Vall d'Hebron Research Institute · Barcelona, Catalonia · NCT06213402

• Evaluating a new injection for patients with transfusion-dependent β-thalassemia

  This study is testing a new injection made from patients' own modified stem cells to see if it can help young adults with severe β-thalassemia reduce their need for blood transfusions.

  Recruiting Early Phase 1 Shenzhen Hemogen · Shenzhen, Guangdong · NCT05864170

• Evaluating a new treatment for patients with transfusion-dependent α-thalassemia

  This study is testing a new treatment using patients' own stem cells to see if it can help young people with severe α-thalassemia who need regular blood transfusions.

  Recruiting Early Phase 1 Shenzhen Hemogen · Nanning, Guangxi · NCT05851105

• Evaluating a new treatment for β-thalassemia using modified stem cells

  This study is testing a new treatment for kids with β-thalassemia by using their own modified stem cells to see if it can help improve their condition.

  Recruiting Early Phase 1 Shenzhen Hemogen · Shenzhen, Guangdong · NCT05745532

• Immune suppression treatment for sickle cell disease and beta-thalassemia patients receiving stem cell transplants

  This study is testing if a new immune suppression treatment can help people with sickle cell disease or beta-thalassemia have better outcomes when they receive stem cell transplants.

  Recruiting at 6 sites Phase 2 Memorial Sloan Kettering Cancer Center · Basking Ridge, New Jersey and 5 other locations · NCT05736419

• Evaluating Atrial Fibrillation in Beta-Thalassemia Patients

  This study looks at the differences between beta-thalassemia patients who have atrial fibrillation and those who don’t to better understand their health.

  Recruiting University Hospital of Ferrara · Ferrara · NCT05508932

• 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

• Long-term observation of non-cancer blood disorders

  This study is tracking people with blood disorders like hemophilia and thrombosis for at least 15 years to see how their conditions change over time and gather useful information for doctors and researchers.

  Recruiting at 74 sites American Thrombosis and Hemostasis Network · Phoenix, Arizona and 75 other locations · NCT04398628

NIH-funded research (9)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Iron overload and low oxygen in pregnancy with beta-thalassemia

  A patient-friendly look at how excess iron and low oxygen during pregnancy could affect mothers and babies in beta-thalassemia.

  NIH research University of Florida · Gainesville, United States · NIH-11392553

• New mouse models and gene therapy for severe alpha-thalassemia

  A patient-focused research opportunity to better understand severe alpha-thalassemia and explore gene-therapy approaches using new mouse models.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11355625

• Genome editing approaches to treat alpha thalassemia

  A research project aims to develop new genome-based therapies to help people with alpha-thalassemia, including those with the most severe form.

  NIH research University of California, San Francisco · San Francisco, United States · NIH-11226215

• How excess iron and low oxygen affect pregnancy in people with beta-thalassemia

  Looking at how excess iron and low oxygen in beta-thalassemia affect mothers, placentas, and babies during pregnancy.

  NIH research University of Florida · Gainesville, United States · NIH-11194518

• Creating new mouse models to understand and treat severe alpha-thalassemia.

  This study is creating new mouse models to help us learn more about severe alpha-thalassemia, a condition that affects red blood cells, and to explore possible gene therapy treatments that could help people with this disease.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11143790

• Clearing excess alpha-globin to help people with beta-thalassemia

  This research looks at whether boosting a cell 'self-cleaning' pathway (ULK1-driven autophagy) can remove toxic excess alpha-globin and help people with beta-thalassemia.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11125822

• Effects of iron overload and low oxygen on pregnancy in women with β-thalassemia

  This study looks at how too much iron and not enough oxygen during pregnancy can affect the health of women with β-thalassemia and their babies, aiming to find ways to make pregnancy safer for them.

  NIH research University of Florida · Gainesville, United States · NIH-11049349

• Developing gene therapy to treat alpha thalassemia

  This study is exploring new gene therapy methods to help people with severe alpha thalassemia, a blood disorder that makes it hard for your body to produce healthy hemoglobin, by using cutting-edge techniques to fix the genes that cause the problem, offering hope for a better treatment than the current options like blo

  NIH research University of California, San Francisco · San Francisco, United States · NIH-11004158

• Investigating how a specific protein process affects beta-thalassemia

  This study is looking at how a special protein can help reduce harmful buildup in the blood of people with beta-thalassemia, a condition that affects hemoglobin, and it aims to find new treatment options for patients by testing this in mice.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-10888292

Related topics

Quick answers

How many Thalassemia clinical trials are recruiting right now?

18, 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 21 countries. The most sites are in China (7 trials), United States (5 trials), Spain (3 trials), Saudi Arabia (3 trials), Italy (3 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Early Phase 1: 4, Phase 3: 3, Phase 1: 2, Phase 2: 2. 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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