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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
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