Gene therapy for inherited optic nerve vision loss
Part of Brain & nervous system, Eyes & vision, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a gene therapy injection into the eye to improve vision in people with a specific type of inherited optic nerve disease called Leber’s hereditary optic neuropathy (LHON). It is aimed at people who have a particular genetic change and a history of vision loss lasting long enough to measure change, while checking for safety first.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be between 18 and 75 years old (or between 12 and 75 for a safety/monitoring group).
- Have LHON vision loss in at least one eye, and your vision test result must meet the trial’s minimum level.
- Confirm you have the LHON genetic mutation called G11778A in the ND4 gene, with no other specific LHON mutations.
- The worse-vision eye must have had vision loss for more than 6 months but less than 10 years.
- Agree to consent and follow the study rules, including eye exam needs (your pupils must be able to be dilated for testing).
- If you are male or female who could become pregnant, follow the trial’s pregnancy-prevention and breastfeeding rules for at least 6 months after treatment.
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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