Gene therapy study for vision loss from LHON
Part of Brain & nervous system, Eyes & vision, Genetic & congenital, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 15 years or older when your vision loss started.
- Vision loss in both eyes is due to LHON caused by a change in the ND4 gene.
- Your vision loss in the first affected eye started between 6 months and 1.5 years ago.
- You have had genetic testing confirming the ND4 mutation and no other harmful mutations.
- You have not used idebenone in the last 7 days and are not allergic to the study drug.
- You are not pregnant, breastfeeding, and you agree to use birth control.
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 uses a special eye scan (OCT) to look at the layer of nerve fibers in the retina of people who carry (or don't carry) a specific gene change linked to Leber Hereditary Optic Neuropathy (NOHL). The goal is to find early warning signs in the blood that might show who is at risk of losing vision.
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.
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 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 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.
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.
Hear when a new Leber Hereditary Optic Disease trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.