Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis | Not Recruiting
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
Health Conditions
  • Retinal Degeneration
Not Recruiting
Recruitment Status
NCT00643747
Primary Trial ID Number
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.
Primary Outcome Measures
  • intraocular inflammation; at intervals up to 12 months
Secondary Outcome Measures
  • visual function; intervals up to 12 months
Research Question
  • 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.
Design Type
Sorry, this information is not available
Ethics Approval
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Publications
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Countries of Recruitment
United Kingdom
Participant Sex
Both
Participant Age Range
5 Years to 30 Years
Participant Type
Sorry, this information is not available
Trial Sample Size
12
Participant Inclusion Criteria
  • 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
Participant Exclusion Criteria
  • 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
Interventions
Biological; tgAAG76 (rAAV 2/2.hRPE65p.hRPE65); Single subretinal injection of vector suspension; up to 3x10e12 vector particles; [A]
Design Details
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Study Design
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Results Reporting
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Acronym
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Scientific Title
An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (AAV2/2-hRPE65p-hRPE65) for Gene Therapy of Severe Early-onset Retinal Degeneration
Secondary Trial Identifying Number
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Website
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Study Funded By
University College, London
Funder Type
Sorry, this information is not available
Study Sponsored By
University College, London
Study Also Sponsored By
Moorfields Eye Hospital NHS Foundation Trust; Targeted Genetics Corporation
Primary Sponsor Type
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Secondary Sponsor Type
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Key Dates

Date of First Enrollment
Date Not Available
Recruitment End Date
Date Not Available
Trial End Date
Date Not Available
Date added to Registry

20 Mar 2008

Last Updated

13 Dec 2013

Date Record Refreshed on UKCTG

31 Jul 2015