Gene therapy for severe sickle cell disease
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a new gene therapy called KL003 for people with severe sickle cell disease. The goal is to see if it can reduce painful episodes and improve quality of life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 12 and 50 years old.
- You have sickle cell disease with a severe type (like HbSS or HbSβ0).
- You have had at least 4 severe pain crises in the last 2 years despite pain management.
- You are generally able to care for yourself (performance status score of 60 or higher).
- You have tried or cannot take hydroxyurea, a standard sickle cell medicine.
- You have been followed at the study center for at least 2 years with detailed medical records.
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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