Gene therapy eye surgery for LCA5 vision loss
Part of Eyes & vision clinical trials.
This Phase 1/2 study tests the safety of putting a gene medicine into the eye for people with vision loss caused by LCA5 gene mutations. It includes a treatment group and a separate group of people who do not receive the gene medicine during the study.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) can sign the consent/assent forms and follow study instructions and visits
- Part A: age 13 or older; Part B: age 4 or older and able to do the required child vision testing
- Your genetic test shows disease-causing changes in the LCA5 gene from both parents (biallelic) from a certified lab
- In the eye to be treated, your vision is worse than a specific limit (under 20/80 on an eye chart, with adjustments for low vision)
- Eye scans show working light-sensing cells (photoreceptors) on OCT imaging
- An eye surgeon/investigator believes you are a good candidate for the planned surgery
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 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.
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