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	The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation when vision loss is present for six months or less. (Show More)
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

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

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

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