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Clinical Trial Report

Gene Therapy Trial Report

Summary

Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations


NCTID NCT00821340 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Leber Congenital Amaurosis-Type 2
Disease Ontology Term DOID:0110016
Compound Name RAAV2-hRPE65
Sponsor Hadassah Medical Organization
Funder Type Other
Recruitment Status
Completed
Enrollment Count 3
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 Undisclosed
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2009-01-12
Completion Date 2017-01-01
Last Update 2018-04-10

Participation Criteria


Eligible Age >=8 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations Israel

Regulatory Information


Has US IND False
FDA Designations
Recent Updates Enrolled 3/10 planned patients

Resources/Links