Gene therapy for transfusion-dependent beta-thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a one-time gene therapy using your own stem cells to help you produce healthy red blood cells and reduce or eliminate the need for regular blood transfusions. It is for people with transfusion-dependent beta-thalassemia who are between 3 and 35 years old.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of transfusion-dependent beta-thalassemia (inherited blood disorder) and need at least 10 units of blood transfusions per year or 100 mL per kg of body weight per year for the last 2 years.
- You must be at least 3 years old and no older than 35 years old.
- Your heart, lungs, kidneys, and liver must work well enough to safely undergo stem cell collection and treatment.
- You must have a good performance status (able to do daily activities with minimal help).
- You must agree to use effective contraception for at least 12 months after treatment if you are of reproductive age.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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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.
This early study tests the safety and potential benefit of the LentiRed gene-based drug in people with transfusion-dependent beta-thalassemia (TDT) who are planning an autologous stem cell transplant. It may help reduce the severity of anemia, but the main goal at this stage is to check safety and side effects.
This trial tests a new treatment called KL003 for people with beta-thalassemia who need regular blood transfusions. The treatment uses your own stem cells (collected from your blood) to try to reduce or stop the need for transfusions.
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This trial tests a gene therapy to help people with transfusion-dependent beta-thalassemia (a severe type of thalassemia) stop needing regular blood transfusions. It uses your own stem cells, which are modified to produce healthy red blood cells.
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