CompletedPhase 1/2

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

United States, United Kingdom15 participantsStarted 2016-04

Plain-language summary

A clinical trial of AAV2/5 vector for patients with Defects in RPE65

Who can participate

Age range3 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Key Inclusion Criteria: * Aged 3 years or older * Early-onset severe retinal dystrophy consistent with RPE65 deficiency Key Exclusion Criteria: * Females who are pregnant or breastfeeding * Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.

What they're measuring

Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event

Timeframe: 6 months

Trial details

NCT IDNCT02781480

SponsorMeiraGTx UK II Ltd

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-12

Results submitted2021-03-10

View on ClinicalTrials.gov More Leber Congenital Amaurosis trials