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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 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.

Clinical Trial 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

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02946879
Study type Observational
Source MeiraGTx UK II Ltd
Contact
Status Completed
Phase
Start date November 2016
Completion date July 2023
View Details
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