Extension 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 Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene; MedDRA version: 20.0Level: PTClassification code 10070667Term: Leber's congenital amaurosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Eye Diseases [C11]

• Registration Number: EUCTR2018-003500-40-BE
• Lead Sponsor: ProQR Therapeutics IV B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-02-26
• Last Update Posted: 9 May 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 11

Inclusion Criteria

1.Subjects who completed participation in the study PQ-110-001 and who may derive benefit from continued treatment with QR-110, as assessed by the Investigator, in consultation with the Medical Monitor
2.Persistence of detectable outer nuclear layer (ONL) in the area of the macula in the opinion of the Investigator, as determined by OCT.
3.Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as assessed by the Investigator.
4.An adult (= 18 years) willing and able to provide informed consent for participation OR a minor (6 to < 18 years) with a parent or legal guardian willing and able to provide written permission for the subject’s participation prior to performing any study related procedures, and pediatric subjects able to provide age-appropriate assent for study participation.

Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Any contraindication to IVT injection according to the Investigator’s clinical judgment and international guidelines (Avery 2014).
2.Any ocular or systemic disease or condition (including medications and laboratory test abnormalities) that could compromise subject safety or interfere with assessment of efficacy and safety, as determined by the Investigator.

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 of the study is to evaluate long term safety and tolerability of QR-110 administered via IVT injection in subjects with LCA due to the CEP290 p.Cys998X mutation.;Secondary Objective: The secondary objectives of the study are:<br>* to evaluate long-term and sustained efficacy of QR-110 administered by IVT injection, as assessed by functional and structural outcome measures<br>* to evaluate changes in Patient Reported Outcome (PROs) measures in subjects treated with QR-110<br>* to evaluate long-term serum pharmacokinetics of QR-110 administered by IVT injection<br>;Primary end point(s): - Frequency and severity of - ocular adverse events (AEs)<br>- Frequency and severity of non-ocular AEs;Timepoint(s) of evaluation of this end point: 24 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): * Change from Baseline in BCVA<br>* Change from Baseline in Mobility course score (unilateral and binocular)<br>* Change from Baseline in photoreceptor outer segment layer thickness by optical coherence tomography (OCT) (if applicable)<br>* Change in OCI<br>* Change in Full-FST (blue and red stimuli; white at Investigator discretion) (thresholds)<br>* Change from Baseline in Patient Reported Outcomes<br>* Change in Pupillary Light Reflex (PLR) (latency and amplitude)<br>* Change in Near Infrared AutoFluorescence (NIRAF) <br>* Pharmacokinetics: Characterize the PK profile of QR-110 in serum;Timepoint(s) of evaluation of this end point: 24 months