Leber's Congenital Amaurosis Clinical Trial
Official title:
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
| Verified date | December 2022 |
| Source | ProQR Therapeutics |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 11 |
| Est. completion date | October 2, 2019 |
| Est. primary completion date | October 2, 2019 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 6 Years and older |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| Belgium | Ghent University Hospital and Ghent University | Ghent | |
| United States | University of Iowa | Iowa City | Iowa |
| United States | Scheie Eye Institute, University of Pennsylvania | Philadelphia | Pennsylvania |
| Lead Sponsor | Collaborator |
|---|---|
| ProQR Therapeutics |
United States, Belgium,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes | 1 year | ||
| Secondary | Frequency and Severity of Non-ocular Adverse Events | 1 year | ||
| Secondary | Change in Best-corrected Visual Acuity (BCVA) | 1 year | ||
| Secondary | Change in Full-field Stimulus Test (FST) | Average Red Light Score | 1 year | |
| Secondary | Change in Full-field Stimulus Test (FST) | Average Blue Light Score | 1 year |