HG004 gene therapy for RPE65 vision loss
Part of Eyes & vision clinical trials.
This Phase 1/2 trial tests the safety and early effectiveness of HG004 (a gene therapy) in people with Leber congenital amaurosis caused by RPE65 gene changes. It aims to see whether treating the eye can improve or stabilize vision-related measures while monitoring side effects.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 6 and 50 years old when you sign consent
- Your diagnosis is Leber congenital amaurosis confirmed by an RPE65 gene test
- You can do the study’s vision and retina (back-of-eye) testing
- For the study eye, vision is reduced (≤ 20/80) OR your visual field is very narrow (less than 20 degrees)
- Your blood and urine lab tests are within acceptable ranges
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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