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Clinical Trial Report

Gene Therapy Trial Report

Summary

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation


NCTID NCT02652767 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Leber Hereditary Optic Neuropathy (LHON)
Disease Ontology Term DOID:705
Compound Name LUMEVOQ
Compound Alias GS010
Sponsor GenSight Biologics
Funder Type Industry
Recruitment Status
Completed
Enrollment Count 39
Results Posted View Results

Therapy Information


Target Gene/Variant ND4G11778A
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravitreal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV2
Editor Type none
Dose 1 9E10 vg in 90uL
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase3
Submit Date 2016-01-07
Completion Date 2019-07-04
Last Update 2022-07-29

Participation Criteria


Eligible Age >=15 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 7
Locations United States,Italy,United Kingdom,France,Germany

Regulatory Information


Has US IND True
FDA Designations
Recent Updates EAP due to commence Q3 2024, France only; MAA in UK goal in 2026

Resources/Links