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Autosomal dominant optic atrophy
MONDO:0020250An autosomal dominant hereditary condition characterized by optic atrophy and progressive visual loss.
Also known as: ADOA, DOA, optic atrophy, autosomal dominant, dominant optic atrophy
9 clinical trials for this condition and its sub-types, 6 tagged with Autosomal dominant optic atrophy itself.
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Sub-types of Autosomal dominant optic atrophy
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4 sub-types
- Cerebellar ataxia-areflexia-pes cavus-optic atrophy-sensorineural hearing loss syndrome 0 trials
- Optic atrophy 8 0 trials
- Optic atrophy with or without deafness, ophthalmoplegia, myopathy, ataxia, and neuropathy 0 trials
- Optic atrophy, hearing loss, and peripheral neuropathy, autosomal dominant 0 trials
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Optic atrophy 3 0 trials
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Optic atrophy 5 0 trials
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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