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

Sponsor

MeiraGTx UK II Ltd (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT02946879

Collaborator

Syne Qua Non Limited (Industry)

Enrollment

Locations

76.9

Duration (Months)

13.5

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Leber Congenital Amaurosis (LCA) Eye Diseases Eye Diseases, Hereditary Retinal Diseases	Biological: AAV OPTIRPE65

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 Design

Study Type:

Observational

Anticipated Enrollment :

27 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children With Retinal Dystrophy Owing to Defects in RPE65 (LCA2)

Study Start Date :

Nov 1, 2016

Anticipated Primary Completion Date :

Apr 1, 2023

Anticipated Study Completion Date :

Apr 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Low dose AAV OPTIRPE65 subretinal administration of a single low dose of AAV RPE65	Biological: AAV OPTIRPE65 comparison of different doses of AAV RPE65
Intermediate dose AAV OPTIRPE65 subretinal administration of a single intermediate dose of AAV RPE65	Biological: AAV OPTIRPE65 comparison of different doses of AAV RPE65
High dose AAV OPTIRPE65 subretinal administration of a single highdose of AAV RPE65	Biological: AAV OPTIRPE65 comparison of different doses of AAV RPE65

Arm

Intervention/Treatment

Low dose AAV OPTIRPE65

subretinal administration of a single low dose of AAV RPE65

Biological: AAV OPTIRPE65

comparison of different doses of AAV RPE65

Intermediate dose AAV OPTIRPE65

subretinal administration of a single intermediate dose of AAV RPE65

Biological: AAV OPTIRPE65

comparison of different doses of AAV RPE65

High dose AAV OPTIRPE65

subretinal administration of a single highdose of AAV RPE65

Biological: AAV OPTIRPE65

comparison of different doses of AAV RPE65

Outcome Measures

Primary Outcome Measures

Incidence of Adverse Events related to the treatment [5 years]
Safety is defined as the absence of ATIMP-related safety events

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 100 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Kellogg Eye Center, University of Michigan Health	Ann Arbor	Michigan	United States	48105
2	Moorfields Eye Hospital NHS Foundation Trust	London		United Kingdom