ong term follow-up study of gene therapy trial for LCA2 OPTIRPE65

Phase 1

• Conditions: eber Congenital Amaurosis (LCA) caused by mutations in RPE65; 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: EUCTR2016-000898-20-GB
• Lead Sponsor: MeiraGTx UK Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-01-04
• Last Update Posted: 20 July 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 27

Inclusion Criteria

Inclusion in the study will be limited to individuals who meet the following criteria:
• Are able to give informed consent or assent, with or without the guidance of their parent/guardian where appropriate
• Were enrolled and treated in the prior open-label, Phase I/II, dose escalation study involving singular intraocular administration of AAV2/5-0PTIRPE65
• The contralateral eye may be untreated or treated with Voretigene Neparvovec.
• Are willing to adhere to the protocol and long-term follow-up
Are the trial subjects under 18? yes
Number of subjects for this age range: 9
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary research objective is to assess the longer term safety of AAV2/5-OPTIRPE65 administered to participants in the OPTIRPE65 trial, measured by the presence or absence of adverse events, the assessment of visual acuity, and loss of light perception.;Secondary Objective: The secondary research objective is to explore the longer term efficacy of AAV2/5-OPTIRPE65 in improving visual and retinal function, and quality of life, and protecting against sight impairment from retinal degeneration.;Primary end point(s): The primary study outcome is the safety of the subretinal administration of AAV2/5-OPTIRPE65.;Timepoint(s) of evaluation of this end point: At 9, 12, 18, 24, 36, 48 and 60 months after AAV2/5-OPTIRPE65 administration

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): The secondary outcomes are measures of the efficacy of AAV2/5-OPTIRPE65:<br>1) Any improvements in visual function from baseline that are greater than the test-retest variation and are sustained for at least two consecutive assessments.<br>2) Quality of life will be measured by the Impact of Visual Impairment (IVI) questionnaire and the EQ5D-5L and EQ-5D-Y.;Timepoint(s) of evaluation of this end point: At 9, 12, 18, 24, 36, 48 and 60 months after AAV2/5-OPTIRPE65 administration