CompletedNot Applicable

Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65

United States, United Kingdom37 participantsStarted 2016-04

Plain-language summary

MGT005 is a natural history study to collect longitudinal prospective data from patients with Leber Congenital Amaurosis associated 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.

Inclusion Criteria: * Patients with RPE65 associated retinal dystrophy * Minimum subject age of 3 years * Able to give consent/parent or guardian able to give consent Exclusion Criteria: * Patients unable or unwilling to undertake consent or clinical testing * Have received a gene therapy treatment in both eyes

What they're measuring

Analysis of retinal structure and function

Timeframe: 6 years

Trial details

NCT IDNCT02714816

SponsorMeiraGTx UK II Ltd

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2023-07-22

View on ClinicalTrials.gov More Leber Congenital Amaurosis trials