Study to evaluate QR-110 in subjects with Leber’s Congenital Amaurosis (LCA) due to the p.Cys998X mutation in the CEP290 Gene

Phase 1

• Conditions: eber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene; MedDRA version: 19.1Level: PTClassification code 10070667Term: Leber's congenital amaurosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2017-000813-22-BE
• Lead Sponsor: ProQR Therapeutics

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-04-13
• Last Update Posted: 16 December 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

1. 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.
2. 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.
3. Detectable outer nuclear layer (ONL) in the area of the macula.
4. An ERG result consistent with LCA.
5. Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging
Are the trial subjects under 18? yes
Number of subjects for this age range: 6
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

1. Syndromic disease
2. Pregnant or breast-feeding female
3. Any clinically significant cardiac disease or defect.
4. One or more coagulation parameters outside of the normal range.
5. Any ocular disease or condition that could compromise treatment safety, visual acuity or interfere with assessment of efficacy and safety.
6.Prior receipt of intraocular surgery or IVT injection within 3 months prior to study start or planned intraocular surgery or procedure during the course of the study
7. 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.
8. Any prior receipt of genetic therapy for LCA

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Secondary Objective: The secondary objectives of the study are:<br>• to evaluate the pharmacokinetics of QR-110 <br>• to evaluate the efficacy of QR-110 <br>;Primary end point(s): Frequency and severity of ocular AEs in the treatment and contralateral eyes ;Timepoint(s) of evaluation of this end point: All 21 visits;Main Objective: The primary objective of the study is to evaluate the safety and tolerability of QR-110 administered via IVT injection in subjects with LCA due to the CEP290 p.Cys998X mutation