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

April 10, 2024 updated by: MeiraGTx UK II Ltd

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)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Actual)

15

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom
        • Moorfields Eye Hospital NHS Foundation Trust
  • United States
    • Michigan
      • Ann Arbor, Michigan, United States, 48105
        • Kellogg Eye Center, University of Michigan Health

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 100 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The patient population are those with Leber Congenital Amaurosis (LCA) with gene mutation RPE65 who have participated in the OPTIRPE65 trial.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Adverse Events related to the treatment
Time Frame: 5 years
Safety is defined as the absence of ATIMP-related safety events
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Improvement in the retinal function
Time Frame: 5 years
Improvements in visual function as assessed by visual assessment
5 years
Improvement in the visual function
Time Frame: 5 years
Improvements in retinal function as assessed by visual assessment
5 years
Improvement in quality of life
Time Frame: 5 years
Improvement in the participant's quality of life which is measurable by QoL questionnaire
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

MeiraGTx UK II Ltd

Collaborators

Syne Qua Non Limited

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2016

Primary Completion (Actual)

July 1, 2023

Study Completion (Actual)

July 1, 2023

Study Registration Dates

First Submitted

July 22, 2016

First Submitted That Met QC Criteria

October 25, 2016

First Posted (Estimated)

October 27, 2016

Study Record Updates

Last Update Posted (Actual)

April 11, 2024

Last Update Submitted That Met QC Criteria

April 10, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MGT004
  • 2016-000898-20 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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