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Leber congenital amaurosis
MONDO:0018998Leber congenital amaurosis (LCA) is a retinal dystrophy defined by blindness and responses to electrophysiological stimulation (Ganzfeld electroretinogram (ERG)) below threshold, associated with severe visual impairment within the first year of life.
Also known as: Leber congenital amaurosis, amaurosis congenita of Leber, Leber's congenital tapetoretinal degeneration, Leber's congenital tapetoretinal dysplasia, congenital absence of the rods and cones, congenital retinal blindness
37 clinical trials for this condition and its sub-types, 10 tagged with Leber congenital amaurosis itself.
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Browse by category →Sub-types of Leber congenital amaurosis
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Leber congenital amaurosis 10 2 trials
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Leber congenital amaurosis 2 2 trials
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Leber congenital amaurosis 5 2 trials
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Leber congenital amaurosis 1 1 trial
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Leber congenital amaurosis 11 0 trials
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Leber congenital amaurosis 12 0 trials
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Leber congenital amaurosis 13 0 trials
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Leber congenital amaurosis 14 0 trials
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Leber congenital amaurosis 15 0 trials
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Leber congenital amaurosis 16 0 trials
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Leber congenital amaurosis 17 0 trials
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Leber congenital amaurosis 18 0 trials
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Leber congenital amaurosis 19 0 trials
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Leber congenital amaurosis 3 0 trials
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Leber congenital amaurosis 4 0 trials
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Leber congenital amaurosis 6 0 trials
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Leber congenital amaurosis 7 0 trials
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Leber congenital amaurosis 8 0 trials
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Leber congenital amaurosis 9 0 trials
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Retinal aplasia 0 trials
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Experimental eye drug hopes to restore sight in rare blindness
Disease control Recruiting nowThis phase 3 trial tests an experimental drug called sepofarsen in 32 people with Leber congenital amaurosis (LCA), a rare genetic condition that causes severe vision loss from birth. The drug is injected into one eye, while the other eye gets a placebo, to see if it safely impro…
Phase 3 • Sponsor: Laboratoires Thea • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Thousands join fight against blindness by sharing their stories
Knowledge-focused Recruiting nowThis registry collects information from people with inherited retinal diseases, like retinitis pigmentosa and Stargardt disease. Participants share their symptoms, family history, and genetic test results online. The goal is to help researchers understand these rare diseases and …
Sponsor: Foundation Fighting Blindness • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:36 UTC