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.
Similar studies
Other trials that look related to this one.
This study tests whether nicotinamide (vitamin B3) can slow or improve vision loss in people with a specific form of LHON. You may be able to join if your symptoms began recently and you have not taken certain other treatments.
This trial tests a new gene therapy called ZVS203e for people with retinitis pigmentosa caused by a specific RHO gene mutation. It aims to see if the treatment is safe and can help improve vision.
This trial tests an injection (PUMCH-E111) for people with a specific genetic form of inherited retinal dystrophy caused by RLBP1 mutations. It aims to see if the treatment is safe and can help preserve vision.
This trial tests a gene therapy called GS010 given as an injection into the eye for people with vision loss from LHON. It aims to see if it can help improve vision in both eyes.
This trial tests an experimental gene therapy (OPGx-RDH12-1001) injected under the retina to treat a rare form of inherited vision loss called LCA caused by RDH12 gene mutations. It aims to see if the treatment is safe and tolerable for people with advanced vision loss.
This trial tests a new gene therapy called VG901, given as an injection into the eye, for people with retinitis pigmentosa caused by a specific genetic change in the CNGA1 gene. The goal is to see if it is safe and tolerable.
Hear when a new Leber's Hereditary Optic Neuropathy (LHON) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.