Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

Recruiting

• Source

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  Public Title	Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
  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
  Acronym	Sorry, not currently available
  Primary Trial Identifying Number	NCT00643747
  Secondary Identifying Number	Sorry, not currently available
  Source of Record	Information obtained from ClinicalTrials.gov on April 14, 2013
  Source of Record URL	http://clinicaltrials.gov/show/NCT00643747
  Date of Registration	2008-03-20
  Date Last Updated	2010-07-07
  Date Record Refreshed on UKCTG	2013-04-16

• Trial

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  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. (from ClinicalTrials.gov)
  Ethics Approval	Sorry, not currently available
  Study Design	Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
  Design Type	Sorry, not currently available
  Design Details	Sorry, not currently available
  Health Condition(s) or Problem	Retinal Degeneration
  Participants 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
  Participants 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
  Participant Sex	Sorry, not currently available
  Participant Age Range	Sorry, not currently available
  Participant Type	Sorry, not currently available
  Target Sample Size	12
  Date of First Enrolment	Sorry, not currently available
  Date of Recruitment End	Sorry, not currently available
  Date of End of Follow-up	Sorry, not currently available
  Trial End Date	Sorry, not currently available
  Recruitment Status	Recruiting
  Overall Trial Status	Sorry, not currently available
  Countries of Recruitment	United Kingdom
  Nation	England
  Location	London
  Interventions	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 Outcome Measures	intraocular inflammation
  Secondary Outcome Measures	visual function; intervals up to 12 months; Yes
  Website	Sorry, not currently available

• Results

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  Results Reporting	Sorry, not currently available
  Publications	Sorry, not currently available

• 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

• Sponsor

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  Study sponsored by	University College, London
  Study also sponsored by	Moorfields Eye Hospital NHS Foundation Trust; Targeted Genetics Corporation
  Primary Sponsor Type	Sorry, not currently available
  Secondary Sponsor Type	Sorry, not currently available

• Funder

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  Study funded by	University College, London
  Funder Type	Sorry, not currently available