Testing a gene-based treatment for transfusion-dependent thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your legal guardian) can understand the study and sign consent willingly
- You are between 5 and 35 years old
- You have transfusion-dependent beta-thalassemia with specific genetic types, and you have needed regular blood transfusions with persistent very low hemoglobin
- You are expected to receive an autologous stem cell transplant (your own stem cells) after being evaluated by the study doctor
- You have been followed in a specialized thalassemia/record-keeping center for at least 2 years
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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