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
36 clinical trials for this condition and its sub-types.
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Broader categories
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Can a single injection restore sight in a rare childhood blindness?
Cure OngoingThis trial tests a gene therapy called ATSN-101, given as a one-time injection under the retina, for people with Leber congenital amaurosis caused by GUCY2D gene mutations. The goal is to see if the treatment is safe and can improve vision. Participants receive the therapy in one…
Phase 1/2 • Sponsor: Atsena Therapeutics Inc. • Aim: Cure
Last updated Aug 05, 2026 00:00 UTC
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Can a single eye injection restore sight in genetic blindness?
Cure OngoingThis trial tests an experimental gene therapy called OCU400 in people with retinitis pigmentosa or Leber congenital amaurosis, which are inherited conditions that cause progressive vision loss and can lead to blindness. The therapy is given as a single injection into the eye, wit…
Phase 1/2 • Sponsor: Ocugen • Aim: Cure
Last updated Aug 02, 2026 00:00 UTC
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Can a One-Time gene therapy preserve sight for years? a 15-Year study aims to find out
Disease control OngoingThis study follows people who previously received a one-time gene therapy injection for a rare inherited eye disease caused by RPE65 mutations. Researchers will test vision and light sensitivity over up to 15 years to see how well the treatment holds up. The goal is to understand…
Sponsor: Genentech, Inc. • Aim: Disease control
Last updated Aug 21, 2026 00:00 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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Talking it out: therapy group aims to ease parents' stress in rare blindness condition
Symptom relief OngoingThis study tests whether group psychotherapy can help parents of children with Leber congenital amaurosis (LCA) feel better emotionally and handle stress. Forty parents will take part, with half joining therapy groups and the other half serving as a comparison. Researchers will m…
Sponsor: Instituto de Genética Ocular • Aim: Symptom relief
Last updated Jun 27, 2026 13:05 UTC