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NCT02946879 Clinical Trial

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

Eye Diseases Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT02946879
Other study ID # MGT004
Secondary ID 2016-000898-20
Status Completed
Phase
First received
Last updated
Start date November 2016
Est. completion date July 2023

Study information
Verified date April 2024
Source MeiraGTx UK II Ltd
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Completed
Enrollment 15
Est. completion date July 2023
Est. primary completion date July 2023
Accepts healthy volunteers No
Gender All
Age group 3 Years to 100 Years
Eligibility 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

Related Conditions & MeSH terms

Intervention

Biological:
• AAV OPTIRPE65
comparison of different doses of AAV RPE65

Locations
Country Name City State
United Kingdom Moorfields Eye Hospital NHS Foundation Trust London
United States Kellogg Eye Center, University of Michigan Health Ann Arbor Michigan

Sponsors (1)
Lead Sponsor Collaborator
MeiraGTx UK II Ltd

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome
Type Measure Description Time frame Safety issue
Primary Incidence of Adverse Events related to the treatment Safety is defined as the absence of ATIMP-related safety events 5 years
Secondary Improvement in the retinal function Improvements in visual function as assessed by visual assessment 5 years
Secondary Improvement in the visual function Improvements in retinal function as assessed by visual assessment 5 years
Secondary Improvement in quality of life Improvement in the participant's quality of life which is measurable by QoL questionnaire 5 years
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