Find a clinical trial.
Current studies for inherited retinal diseases around the world, gathered every day from ClinicalTrials.gov, the official U.S. registry.
Joining a trial is a big decision. Bring any study you are interested in to your eye doctor. Each study decides for itself who can take part.
To print, tick Add to print list on the studies you want, then press Print selected. Each study prints on its own page with who can join and how to contact the study team.
No trial for your gene change?
Some people have a gene change so rare that only a handful of people in the world share it. A few groups now design a medicine for one person at a time.
The n-Lorem Foundation is a nonprofit that makes personalized antisense oligonucleotide (ASO) medicines, short pieces of genetic material designed to fix how one specific gene change is read. Treatment is free, for life. In 2026 it began treating a person with a PRPH2 retinal dystrophy (NCT07177196).
- The same gene change must be found in only about 1 to 30 people worldwide.
- The genetic cause must be confirmed, and you must live in the United States.
- Your doctor applies on your behalf, and n-Lorem decides whether an ASO could work for your specific change. Many changes cannot be treated this way.
If you think this might apply to you, bring your genetic test report to your retina specialist and ask about it.
What do trial phases mean?
- Phase 1: the first studies in people, usually small. The main goal is safety and finding the right dose.
- Phase 2: a larger group, to learn more about safety and whether the treatment seems to work.
- Phase 3: large studies that compare the treatment with standard care or a placebo. Results are used to seek approval.
- Observational or natural history studies: no treatment is given. Researchers follow how a condition changes over time. These studies are essential for designing future treatment trials.
About this data.
Study details come directly from ClinicalTrials.gov and are written by each study's sponsor. Information can be out of date. Always confirm details with the study team listed on ClinicalTrials.gov. Listing a study here is not an endorsement.