CompletedNot Applicable

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

United States, United Kingdom15 participantsStarted 2016-11

Plain-language 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.

Who can participate

Age range3 Years – 100 Years

SexALL

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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.

What they're measuring

Incidence of Adverse Events related to the treatment

Timeframe: 5 years

Trial details

NCT IDNCT02946879

SponsorMeiraGTx UK II Ltd

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2023-07

View on ClinicalTrials.gov More Leber Congenital Amaurosis (LCA) trials