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

Plain-language summary

This study aimed to provide long-term follow-up of patients who had previously received AAV OPTIRPE65 gene therapy for Leber congenital amaurosis caused by RPE65 defects. It included people who had been enrolled and treated in the earlier Phase I/II dose-escalation study. It was a non-phase follow-up study and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

MeiraGTx UK II Ltd

Intervention

AAV OPTIRPE65

Start date

11/1/2016

Estimated completion

7/1/2023

Countries

United States, United Kingdom

Related genes

Locations

• Ann Arbor, United States
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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.