Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

Completed

• Conditions: Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Leber Congenital Amaurosis (LCA)
• Interventions: Biological: AAV OPTIRPE65

• Registration Number: NCT02946879
• Lead Sponsor: MeiraGTx UK II Ltd

Brief Summary

This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.

Detailed Description

The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.

Study Timeline

• Study First Submitted: 2016-07-22
• Study First Submitted QC: 2016-10-25
• Study First Posted: 2016-10-27
• Study Start: 2016-11
• Primary Completion: 2023-07
• Study Completion: 2023-07
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-10
• Last Update Posted: 2024-04-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

• Were enrolled and treated in the prior open-label, Phase I/II, dose escalation study involving intraocular administration of AAV2/5-OPTIRPE65

Exclusion Criteria

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Low dose AAV OPTIRPE65	AAV OPTIRPE65	subretinal administration of a single low dose of AAV RPE65
Intermediate dose AAV OPTIRPE65	AAV OPTIRPE65	subretinal administration of a single intermediate dose of AAV RPE65
High dose AAV OPTIRPE65	AAV OPTIRPE65	subretinal administration of a single highdose of AAV RPE65

Primary Outcome Measures

Name	Time	Method
Incidence of Adverse Events related to the treatment	5 years	Safety is defined as the absence of ATIMP-related safety events

Secondary Outcome Measures

Name	Time	Method
Improvement in the retinal function	5 years	Improvements in visual function as assessed by visual assessment
Improvement in the visual function	5 years	Improvements in retinal function as assessed by visual assessment
Improvement in quality of life	5 years	Improvement in the participant's quality of life which is measurable by QoL questionnaire

Trial Locations

Locations (2)

Kellogg Eye Center, University of Michigan Health; 🇺🇸 Ann Arbor, Michigan, United States

Moorfields Eye Hospital NHS Foundation Trust; 🇬🇧 London, United Kingdom