CompletedPhase 1/2

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

United Kingdom12 participantsStarted 2007-01

Plain-language summary

The purpose of the study is to determine whether gene therapy is safe and effective for the treatment of severe childhood blindness caused by mutations in RPE65.

Who can participate

Age range5 Years – 30 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: * Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65 Exclusion Criteria: * Visual acuity in the study eye better than 6/36 Snellen * Hypertension * Diabetes mellitus * Tuberculosis * Renal impairment * Immunocompromise * Osteoporosis * Gastric ulceration * Severe affective disorder) * Pregnancy or lactation

What they're measuring

intraocular inflammation

Timeframe: at intervals up to 12 months

Trial details

NCT IDNCT00643747

SponsorUniversity College, London

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2014-12

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