Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

• Status: Completed
• Trial end date: 2019-10-02
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: ProQR Therapeutics

Criteria

Inclusion Criteria:

• Male or female, ≥ 6 years of age at Screening with a clinical diagnosis of LCA and a molecular diagnosis of homozygosity or compound heterozygosity for the CEP290 p.Cys998X mutation.

• Best-corrected visual acuity greater than or equal to light perception in both eyes and equal to or worse than LogMAR +1.0 (Snellen notation 20/200) in the worse eye and equal to or worse than LogMAR +0.7 (Snellen notation 20/100) in the contralateral eye.

• Detectable outer nuclear layer (ONL) in the area of the macula.

• An electroretinogram (ERG) result consistent with LCA.

• Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging.

Exclusion Criteria:

• Syndromic disease.

• Pregnant or breast-feeding female.

• Any clinically significant cardiac disease or defect.

• One or more coagulation parameters outside of the normal range.

• Any ocular disease or condition that could compromise treatment safety, visual acuity or interfere with assessment of efficacy and safety.

• Prior receipt of intraocular surgery or intravitreal injection within 3 months prior to study start or planned intraocular surgery or procedure during the course of the study.

• Use of any investigational drug or device within 90 days or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the PQ-110-001 study period.

• Any prior receipt of genetic therapy for LCA