Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

Recruiting

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  • Source

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    Public Title Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
    Acronym Sorry, not currently available
    Source of Record URL http://clinicaltrials.gov/show/NCT00643747

  • Trial

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    Health Condition(s) or Problem Retinal Degeneration
    Lay 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. (from ClinicalTrials.gov)
    Who can enter the trial 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
    Who cannot enter the trial 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 will happen Biological; tgAAG76 (rAAV 2/2.hRPE65p.hRPE65); Single subretinal injection of vector suspension; up to 3x10e12 vector particles; A; rAAV 2/2.hRPE65p.hRPE65
    Primary aim intraocular inflammation
    Secondary Aim visual function; intervals up to 12 months; Yes
    Participant Information Sheet Sorry, not currently available
    Website Sorry, not currently available
    Recruitment Status Recruiting
    Nation England
    Location London

  • Contact

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    Contact for Public Queries James WB Bainbridge, PhD FRCOphth 02076084023 mol.therapy@ucl.ac.uk Robin R Ali, PhD Study Director University College, London
    Contact for Scientific Queries James WB Bainbridge, PhD FRCOphth; Principal Investigator
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