Condition
Transfusion-dependent Beta-Thalassemia clinical trials and research
As of November 12, 2026, Find a Trial lists 5 Transfusion-dependent Beta-Thalassemia clinical trials that are recruiting, at sites in 3 countries (most in China (3), Italy (1), United States (1)). By phase: Early Phase 1 (1), Phase 2 (1), Phase 1/2 (1), Phase 3 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 5Clinical trials
- 3Countries
Newest Transfusion-dependent Beta-Thalassemia clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Gene-modified stem cell therapy for transfusion-dependent beta-thalassemia | Phase 2 | Rome, Lazio and 1 other locations | Recruiting | Jul 2, 2026 |
| Mitapivat for pediatric transfusion-dependent thalassemia (α- or β-TDT) | Phase 3 | Atlanta, Georgia and 3 other locations | Recruiting | Apr 2, 2026 |
| Treatment for Beta-Thalassemia using KL003 Cell Injection | Phase 1/2 | Shanghai, Shanghai Municipality and 1 other locations | Recruiting | Feb 28, 2024 |
| Comparing growth and development in children with beta-thalassemia after gene therapy | — | Tianjin, Tianjin Municipality | Recruiting | Aug 14, 2023 |
| Evaluating a new gene therapy for transfusion-dependent beta-thalassemia | Early Phase 1 | Nanning, Guangxi | Recruiting | Mar 9, 2023 |
All recruiting trials (5)
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Gene-modified stem cell therapy for transfusion-dependent beta-thalassemia
A small, early-phase study will test a one-time infusion of a patient’s own genetically edited stem cells to see if it reduces transfusion needs in adults and children with transfusion-dependent beta-thalassemia.
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Mitapivat for pediatric transfusion-dependent thalassemia (α- or β-TDT)
This pediatric trial tests whether mitapivat can reduce or lessen the need for transfusions in children with transfusion-dependent alpha- or beta-thalassemia.
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Treatment for Beta-Thalassemia using KL003 Cell Injection
This study is testing a new gene therapy using KL003 cell injections to see if it can help young people with severe beta-thalassemia reduce their need for blood transfusions.
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Comparing growth and development in children with beta-thalassemia after gene therapy
This study looks at how gene therapy affects the growth and development of children with beta-thalassemia compared to those getting regular treatments and healthy kids.
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Evaluating a new gene therapy for transfusion-dependent beta-thalassemia
This study is testing a new gene therapy to see if it can help people with transfusion-dependent beta-thalassemia reduce or stop their need for regular blood transfusions.
Related topics
Quick answers
How many Transfusion-dependent Beta-Thalassemia clinical trials are recruiting right now?
5, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 3 countries. The most sites are in China (3 trials), Italy (1 trial), United States (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Early Phase 1: 1, Phase 2: 1, Phase 1/2: 1, Phase 3: 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.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.