Clinical trials
Leber Congenital Amaurosis clinical trials
Below are recruiting leber congenital amaurosis clinical trials, each written for real people, not researchers. We’re tracking 4 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT05906953RecruitingPhase 1/Phase 2
HG004 gene therapy for RPE65 vision loss
This Phase 1/2 trial tests the safety and early effectiveness of HG004 (a gene therapy) in people with Leber congenital amaurosis caused by RPE65 gene changes. It aims to see whether treating the eye can improve or stabilize vision-related measures while monitoring side effects.
Sacramento, CaliforniaAges 6–50 - NCT06891443RecruitingPhase 3
Study of Sepofarsen for LCA type 10
This study tests an investigational RNA therapy called sepofarsen for people with Leber Congenital Amaurosis (LCA) type 10, a rare inherited eye disease that causes severe vision loss from birth. The goal is to see if it can slow or improve vision loss.
San Francisco, CaliforniaAges 6 years+ - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age - NCT02435940Recruiting
Registry for inherited eye vision conditions
This study is a registry, meaning it collects information about people with inherited retinal (retina) diseases. It may help researchers learn how these conditions progress and connect you with future studies or treatments.
Columbia, MarylandAges Any age
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Common questions
- Are there clinical trials for leber congenital amaurosis?
- Yes. Clin2 currently lists 4 recruiting leber congenital amaurosis studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a leber congenital amaurosis trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a leber congenital amaurosis trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.