ProQR Leber Congenital Amaurosis (CEP290) RNA Therapy Trial
PQ-110-003
The purpose of this study is to learn about QR-110, a new trial medication for patients with Leber’s Congenital Amaurosis caused by the CEP290 mutation. This study is being done to find out if the study drug works and is safe in patients with LCA10. The study drug is delivered by intravitreal injection. Main eligibility criteria:
- Must be at least 8 years old.
- Must have CEP290-related retinal degeneration caused by a molecular diagnosis of homozygosity or compound heterozygosity for the CEP290 p.Cys998X mutation.
- Must have visual acuity no better than 20/50 in one eye, but no worse than hand motion.
- 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 2 years (25 months). There are 21 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. If you are interested in learning more about this study or have a patient that you believe would be a good candidate, please call the study coordinator at 503-494-0020 or email ordc@ohsu.edu.
Principal Investigator:
Paul Yang, MD, PhD.
Casey Eye Institute
3375 SW Terwilliger Blvd.
Portland, OR 97239
eIRB#: 19715