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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