Condition
Leber Congenital Amaurosis clinical trials and research
As of November 12, 2026, Find a Trial lists 4 Leber Congenital Amaurosis clinical trials that are recruiting, at sites in 10 countries (most in United States (4), Australia (1), Belgium (1)). By phase: Phase 1/2 (1), Phase 3 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 4Clinical trials
- 10Countries
Newest Leber Congenital Amaurosis clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Safety and tolerability of a single retina gene therapy for RDH12-related LCA | Phase 1/2 | Phoenix, Arizona and 3 other locations | Recruiting | Jul 2, 2026 |
| Sepofarsen injections for LCA10 due to the CEP290 p.Cys998X mutation | Phase 3 | San Francisco, California and 17 other locations | Recruiting | Mar 24, 2025 |
| Registry for inherited retinal degenerative diseases | — | Columbia, Maryland | Recruiting | May 6, 2015 |
| Registry for patients with rare diseases | — | Sioux Falls, South Dakota | Recruiting | Feb 15, 2013 |
All recruiting trials (4)
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Safety and tolerability of a single retina gene therapy for RDH12-related LCA
This early-stage gene therapy test tries a one-time retinal injection of OPGx-RDH12 to see if it is safe and may help people with RDH12-related Leber congenital amaurosis.
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Sepofarsen injections for LCA10 due to the CEP290 p.Cys998X mutation
This study will test whether sepofarsen eye injections can improve vision in children and adults with LCA10 caused by the CEP290 c.2991+1655A>G (p.Cys998X) mutation.
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Registry for inherited retinal degenerative diseases
This study is creating a registry for people with rare inherited eye diseases to help track their health and experiences over time, making it easier to understand these conditions and support research.
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Registry for patients with rare diseases
This study is creating a central registry for people with rare diseases to help connect them with researchers and improve treatments.
Related topics
Quick answers
How many Leber Congenital Amaurosis clinical trials are recruiting right now?
4, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 10 countries. The most sites are in United States (4 trials), Australia (1 trial), Belgium (1 trial), Brazil (1 trial), Canada (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 1/2: 1, Phase 3: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.