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Clinical Trial Report

Gene Therapy Trial Report

Summary

Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65


NCTID NCT01496040 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Leber Congenital Amaurosis
Disease Ontology Term DOID:0110016
Compound Name RAAV2/4.hRPE65
Sponsor Nantes University Hospital
Funder Type Other
Recruitment Status
Completed
Enrollment Count 9
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 AAV2/4
Editor Type
Dose 1 1.22E10 -2E10 vg
Dose 2 3.27E10-4.8E10 vg
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2011-11-24
Completion Date 2014-08
Last Update 2015-10-07

Participation Criteria


Eligible Age 6 Years - 50 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND
FDA Designations
Recent Updates Clinical study demonstrated safety, inconsistent findings on long-term efficacy

Resources/Links