Gene therapy trial for children with BBS1 retinal degeneration
Part of Eyes & vision clinical trials.
This early-phase trial tests a gene therapy called AXV-101 for children with vision loss caused by BBS1 genetic mutations. The goal is to see if it can help slow or stop retinal damage.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Aged 4 to 17 years old.
- Must have a confirmed BBS1 genetic mutation (two copies, one from each parent).
- Show signs of retinal degeneration, like trouble seeing at night or cone-rod dystrophy.
- Have enough healthy retinal cells measured by a special eye scan (OCT).
- Parent or guardian must agree to the trial and follow study visits.
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 trial tests a gene therapy called OPGx-BEST1 injected under the retina to treat vision loss from Best vitelliform macular dystrophy or a related condition. It may help people with advanced disease who have a specific genetic change in the BEST1 gene.
This trial tests a new gene therapy for Bietti's crystalline dystrophy, a rare inherited eye disease that causes progressive vision loss. The treatment aims to slow or stop vision loss by delivering a healthy gene to the eye.
This trial tests a gene therapy called ATSN-201 for people with X-linked retinoschisis (XLRS), a condition that affects the retina and can cause vision loss. The therapy aims to fix the faulty gene that causes the condition, potentially halting vision loss and improving vision. Participants receive a one-time injection into the eye, and the study monitors safety and effectiveness.
This trial tests a single gene therapy injection under the retina for boys with X-linked retinoschisis, a condition that causes vision loss. It aims to see if the treatment is safe and can improve vision.
This Phase 1/2 study tests a gene therapy treatment (BS01) in people with retinitis pigmentosa to try to improve or preserve vision. You may be a candidate if your vision in at least one eye is quite limited and you haven’t had certain gene therapies before.
This trial tests a new gene therapy called ZVS106e for people with inherited retinal degeneration caused by changes in both copies of the ABCA4 gene. The therapy aims to slow or stop vision loss, and the study will check if it is safe and if it works.
Hear when a new Retinal Degeneration trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.