Phase 2
N=11
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
Leber's Congenital Amaurosis
Bottom Line
View on ClinicalTrials.gov: NCT03140969 ↗Enrolled (actual)
11
Serious AEs
54.6%
Results posted
Dec 2022
Primary outcome: Primary: Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes — 6; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- QR-110 (Drug)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- All
- Sponsor
- Laboratoires Thea
- Primary completion
- Oct 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes |
6; 5 | — |
| SECONDARY Frequency and Severity of Non-ocular Adverse Events |
6; 5 | — |
| SECONDARY Change in Best-corrected Visual Acuity (BCVA) |
2.597; 2.990; -0.813; -0.126; -0.903; -0.122 | — |
| SECONDARY Change in Full-field Stimulus Test (FST) |
-0.6; -0.9 | — |
| SECONDARY Change in Full-field Stimulus Test (FST) |
-0.6; -0.9 | — |
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.
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT03140969). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.