Study to evaluate the efficacy, safety, tolerability and the extent to which the study drug is distributed in the body of multiple doses of QR-110 in subjects with LCA10 compared to a sham procedure. The study will be double-masked, randomized and controlled which means that both patients and study staff will not know and cannot influence who receives which treatment (study drug of sham).
- Conditions
- Therapeutic area: Diseases [C] - Eye Diseases [C11]MedDRA version: 20.0Level: PTClassification code 10070667Term: Leber's congenital amaurosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorderseber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
- Registration Number
- EUCTR2018-003501-25-FR
- Lead Sponsor
- ProQR Therapeutics
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 30
1.Male or female, = 8 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, based on genotyping analysis at Screening. Historic genotyping results from a certified laboratory are acceptable with Sponsor approval.
2.Detectable outer nuclear layer (ONL) in the area of the macula as determined by the reading center at Screening.
3.An ERG result consistent with LCA, as determined by the reading center. A historic ERG result may be acceptable for eligibility
4.Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as assessed by the Investigator.
Are the trial subjects under 18? yes
Number of subjects for this age range: 13
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 17
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1.Any contraindication to IVT injection according to the Investigator’s clinical judgment and international guidelines (Avery 2014)
2.Any ocular and/or general disease or condition that could compromise subject’s safety or interfere with assessment of efficacy and safety, as determined by the Investigator
3.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
4.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 110-003 study period
5.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 Main Objective: The primary objective is to evaluate the efficacy of QR-110 administered by intravitreal (IVT) injection in subjects with LCA due to the CEP290 p.Cys998X mutation<br><br>;Secondary Objective: The secondary objectives are to evaluate:<br>- The safety and tolerability of QR-110 administered via IVT injection in subjects with LCA due to the CEP290 p.Cys998X mutation <br>- Changes in quality of life in subjects with LCA due to the CEP290 p.Cys998X mutation treated with QR-110 <br>- The systemic exposure of QR-110 administered by IVT injection in subjects with LCA due to the CEP290 p.Cys998X mutation<br>;Primary end point(s): The primary endpoint is mean change in BCVA relative to baseline versus sham;Timepoint(s) of evaluation of this end point: 12 months
- Secondary Outcome Measures
Name Time Method