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
38 clinical trials for this condition and its sub-types, 11 tagged with Leber congenital amaurosis itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Leber congenital amaurosis
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Leber congenital amaurosis 2 3 trials
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Leber congenital amaurosis 10 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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A gene therapy injected into the eye aims to restore vision lost to a rare inherited retinal disease
Cure CompletedResearchers are testing a gene therapy called rAAV2/4.hRPE65 in people with Leber congenital amaurosis or severe early-onset retinal degeneration caused by mutations in the RPE65 gene. The treatment delivers a working copy of the gene directly into one eye through a single inject…
Phase 1/2 • Sponsor: Nantes University Hospital • Aim: Cure
Last updated Sep 16, 2026 00:00 UTC
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One-Time gene injection aims to restore sight in rare childhood blindness
Cure CompletedThis trial tests a gene therapy for people with a rare inherited eye disease called Leber congenital amaurosis, caused by mutations in the RPE65 gene. A harmless virus is used to deliver a working copy of the gene into cells under the retina. The goal is to restore vision with a …
Phase 1 • Sponsor: University of Pennsylvania • Aim: Cure
Last updated Jul 19, 2026 00:00 UTC
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Mapping the slow fade of RPE65 blindness to speed future cures
Knowledge-focused CompletedResearchers are following people with Leber congenital amaurosis caused by RPE65 gene mutations to see how their vision and retinas change over time. The study enrolls children and adults, ages 3 and older, who have this inherited retinal dystrophy. Participants undergo regular e…
Sponsor: MeiraGTx UK II Ltd • Aim: Knowledge-focused
Last updated Sep 21, 2026 14:00 UTC
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Gene therapy safety check: luxturna registry wraps up
Knowledge-focused CompletedThis completed study tracked 87 patients who received Luxturna gene therapy for a rare inherited eye condition that causes blindness. Researchers monitored side effects and pregnancy outcomes for up to 5 years after treatment. The goal was to gather real-world safety data, not to…
Sponsor: Spark Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:51 UTC