Gene therapy for certain RPE65 inherited eye conditions
Part of Eyes & vision clinical trials.
This early-stage trial tests a gene therapy aimed at treating inherited retinal dystrophy caused by changes in the RPE65 gene on both copies. It may help slow vision loss or improve eye function, but the study is mainly about safety and learning how the treatment works.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your guardian) can understand the study and sign the consent form
- You are between 8 and 45 years old when you sign
- You have an inherited retinal condition caused by RPE65 changes on both gene copies
- You do not have other eye problems that could interfere with the results
- You do not have other illnesses or infections that are causing likely or uncontrolled vision loss
- You do not have active or suspected autoimmune disease, and you are not pregnant or breastfeeding
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This early study tests a gene therapy designed for people whose vision problems are caused by an RPGR genetic change. It may help slow or improve the eye disease by treating the underlying cause.
This trial tests a gene therapy for people with a specific genetic form of retinitis pigmentosa (caused by changes in the RPGR gene). It aims to improve vision and slow disease progression.
This trial tests an injection (PUMCH-E111) for people with a specific genetic form of inherited retinal dystrophy caused by RLBP1 mutations. It aims to see if the treatment is safe and can help preserve vision.
This trial tests a new gene therapy called ZVS203e for people with retinitis pigmentosa caused by a specific RHO gene mutation. It aims to see if the treatment is safe and can help improve vision.
This trial tests an injection called PUMCH-E101 for people with vision loss caused by changes in the RDH12 gene. The goal is to see if it is safe and if it can help improve vision.
This experimental gene therapy (ZM-02) is designed for people with advanced retinitis pigmentosa who have very poor vision (counting fingers or worse). It aims to see if the treatment is safe and can improve vision by replacing a faulty gene in the retina.
Hear when a new Biallelic RPE65 Mutation-associated Retinal Dystrophy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.