Gene therapy for children with oculocutaneous albinism type 1
Part of Eyes & vision, Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This trial tests a single injection of JWK010 gene therapy for children aged 5 to 12 with OCA1A albinism. The therapy is given into the eye to try to improve vision, and is meant for those who have not had gene therapy before.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 5 and 12 years old.
- You have a confirmed diagnosis of OCA1A albinism with genetic test showing two TYR gene mutations.
- Your better eye (the one not being treated) has vision no worse than 20/400.
- You have not had gene therapy or cell therapy in the past.
- You do not have any other eye conditions that could affect vision or the injection.
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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