An Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

PQ-110-001

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.

2017-10-16

2019-10-02

2019-10-02

Completed

Early phase I

11

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 11, 'type': 'ACTUAL'}}

2022-12-27