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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis
NCTID
NCT00643747
(View at clinicaltrials.gov)
Description
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.
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Development Status
Inactive
Indication
Leber Congenital Amaurosis-Type 2
Disease Ontology Term
DOID:0110016
Compound Name
TgAAG76
Compound Description
rAAV2/2.hRPE65p.hREP65
Sponsor
University College, London
Funder Type
Other
Recruitment Status
Completed
Enrollment Count
12
Results Posted
Not Available
Therapy Information
Target Gene/Variant
RPE65
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Subretinal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
Dose 1
1E11 vg
Dose 2
1E12 vg
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2008-03-20
Completion Date
2014-12
Last Update
2015-12-07
Participation Criteria
Eligible Age
5 Years - 30 Years
Standard Ages
Child, Adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
United Kingdom
Regulatory Information
Has US IND
FDA Designations
Recent Updates
Vector was originally produced by Targeted Genetics Corporation, clinical trial showed a lack of sustained efficacy
Resources/Links
Clinical Publications
Spectral sensitivity measurements reveal partial success in restoring missing rod function with gene therapy
Effect of gene therapy on visual function in Leber's congenital amaurosis
Long-term effect of gene therapy on Leber's congenital amaurosis
Preclinical Publications
Gene therapy in the second eye of RPE65-deficient dogs improves retinal function
RPE65 gene therapy slows cone loss in Rpe65-deficient dogs
Successful gene therapy in older Rpe65-deficient dogs following subretinal injection of an adeno-associated vector expressing RPE65
Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium