A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

• Status: Active, not recruiting
• Trial end date: 2021-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this double-masked, randomized, controlled, multiple-dose study is to evaluate the efficacy, safety, tolerability and systemic exposure of sepofarsen (QR-110) administered via intravitreal injection in subjects with Leber's Congenital Amaurosis (LCA) due to the CEP290 p.Cys998X mutation after 24 months of treatment

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: ProQR Therapeutics

Criteria

Main Inclusion Criteria:

• 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.

• BCVA greater or equal to Logarithm of the Minimum Angle of Resolution (LogMAR) +3.0, and equal or worse than LogMAR + 0.4 in the treatment eye.

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

• An electroretinogram (ERG) result consistent with LCA. A historic ERG result may be acceptable for eligibility.

Main Exclusion Criteria:

• Any contraindication to IVT injection according to the Investigator's clinical judgment and international guidelines (Avery 2014).

• 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.

• 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.

• 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.

• Any prior receipt of genetic therapy for LCA.