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...
Date First Received: March 20, 2008
Last Updated: July 7, 2010
Verified by: University College, London, July 2010
Clinical Trial Phase: Phase 1/Phase 2 | Start Date: January 2007
Overall Status: Recruiting
Estimated Enrollment: 12
Brief Summary
Official 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”
Condition Keyword(s):
Additional Keyword(s) Provided by Sponsor:
Intervention(s):
Condition MeSH Term(s), Assigned with an Experimental Algorithm:
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.
Study Type: Interventional
Study Design: Control: Uncontrolled, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Study Primary Completion Date: January 2013
Detailed Clinical Trial Description
The main objective of the proposed trial is to determine the safety and efficacy subretinal administration of a recombinant adeno-associated viral vector (rAAV 2/2.hRPE65p.hRPE65) at three different dosage levels in individuals with autosomal recessive severe early-onset retinal degeneration due to mutations in RPE65. We have a comprehensive clinical monitoring plan to investigate the safety and efficacy of vector delivery.
Intervention(s) in this Clinical Trial
- Biological: tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)
- Single subretinal injection of vector suspension; up to 3x10e12 vector particles
Arms, Groups and Cohorts in this Clinical Trial
- Experimental: A
- Injection of vector
Outcome Measures for this Clinical Trial
Primary Measures
- intraocular inflammation
- Time Frame: at intervals up to 12 months
Safety Issue?: Yes
- Time Frame: at intervals up to 12 months
Secondary Measures
- visual function
- Time Frame: intervals up to 12 months
Safety Issue?: Yes
- Time Frame: intervals up to 12 months
Criteria for Participation in this Clinical 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
Gender Eligibility for this Clinical Trial: Both
Minimum Age for this Clinical Trial: 5 Years
Maximum Age for this Clinical Trial: 30 Years
Are Healthy Volunteers Accepted for this Clinical Trial?: No
Clinical Trial Sponsor Information
Lead Sponsor: University College, London
Overall Clinical Trial Officials and Contacts
Robin R Ali, PhD Study Director University College, London
Overall Contact: James WB Bainbridge, PhD FRCOphth 02076084023 mol.therapy@ucl.ac.uk
Related Publications
Citations Reporting Results
Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med. 2008 May 22;358(21):2231-9. Epub 2008 Apr 27.
Additional Information
Information obtained from ClinicalTrials.gov on September 01, 2010
Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00643747
Study ID Number: ALIR1015
ClinicalTrials.gov Identifier: NCT00643747
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
Clinical Trials Authorship and Review
Clinical Trials content is provided directly by the U.S. National Institutes of Health via ClinicalTrials.gov and is not reviewed separately by ClinicalTrialsFeeds.org. Every page of specific clinical trials information contains a unique identifier which can be used to find further details directly from the National Institutes of Health.
