Autosomal dominant optic atrophy, classic form
MONDO:0008134One of the most common forms of hereditary optic neuropathy characterized by progressive bilateral visual loss during the first decade of life, associated with optic disk pallor, visual field and color vision defects.
Also known as: Kjer optic atrophy, autosomal dominant optic atrophy, Kjer type, optic atrophy type 1, Kjer-type optic atrophy, OAK, OPA1, optic atrophy 1, optic atrophy, Kjer type
20 clinical trials for this condition and its sub-types, 3 tagged with Autosomal dominant optic atrophy, classic form itself.
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New eye injection aims to slow genetic blindness
Disease control OngoingThis study tests a new medicine called PYC-001, given as an injection into the eye, for people with a genetic condition that damages the optic nerve (OPA1 optic atrophy). The main goal is to check if the treatment is safe and tolerable. About 18 adults will receive a single dose,…
Phase 1 • Sponsor: PYC Therapeutics • Aim: Disease control
Last updated Sep 19, 2026 00:00 UTC
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Could vitamin B3 save sight in rare optic nerve disease?
Disease control OngoingThis study tests whether high-dose nicotinamide (vitamin B3) is safe and can help people with dominant optic atrophy, a rare genetic disease that slowly damages the optic nerve and causes vision loss. Researchers will give 25 adults 3 grams of nicotinamide daily and monitor for s…
Phase 2/3 • Sponsor: University Hospital, Angers • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC