The purpose of this study is to learn about a new gene therapy for patients with Leber Congenital Amaurosis caused by the CEP290 mutation. This is the first study that aims to treat LCA10 by gene therapy and the study investigators want to find out whether it is safe for use in humans. The gene therapy is given by subretinal injection.
Main eligibility criteria:
- Must be at least 3 years old.
- Must have CEP290-related retinal degeneration caused by a compound heterozygous or homozygous intron 26 c.2991+1655A>G mutation.
- Must have visual acuity no better than 20/50 in each eye, but no worse than light perception.
- Good general health.
This study will occur at the Casey Eye Institute in Portland, OR, and at other sites around the world. Participation in this study will last about 3 years. There are 20 required visits in the study. Each study visit will involve functional vision tests and dilated eye exams. All study procedures
are covered by the sponsor and travel reimbursement is available.
Mark Pennesi, MD, PhD.
Casey Eye Institute
3375 SW Terwilliger Blvd.
Portland, OR 97239
IRB Approved: 6/13/2019