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

Completed

Conditions studied: Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases

In brief

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.

Key facts

Study ID

NCT02946879

Run by

MeiraGTx UK II Ltd

People needed

15

Starts

2016-11-01

Expected to finish

2023-07-01

Last updated by the study team

2024-04-11

Who can join

Age: 3 and older, up to 100. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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

Where it is running

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

Full record on ClinicalTrials.gov

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