NCT03293524 - Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

March 12, 2018

Primary Completion Date

July 23, 2024

Study Completion Date

July 23, 2024

Conditions

Leber Hereditary Optic Neuropathy

Interventions

GENETIC

GS010

GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). GS010 will be administrated via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) as a single baseline intravitreal injection.

DRUG

Placebo

The placebo is a BSS, sterile, apyrogenic solution and used for ocular surgery. The placebo will be administered via intravitreal injection in a volume of 90 μL.

Trial Locations (13)

9000

Universitair Ziekenhuis Gent, Ghent

10029

Icahn School of Medicine at Mount Sinai, New York

11217

Taipei Veterans General Hospital, Taipei

19107

Wills Eye Institute - Ocular Oncology Service, Philadelphia

28034

Hospital Universitario Ramon y Cajal, Madrid

30322

Emory Healthcare - The Emory Clinic, Atlanta

37232

Vanderbilt Eye Institute, Nashville

40139

IRCCS Istituto delle Scienze Neurologiche di Bologna UOC Clinica Neurologica, Bologna

75012

CHNO Les Quinze Vingts, Paris

80045

University of Colorado Health Eye Center, Aurora

91105

Doheny Eye Center UCLA Pasadena, Pasadena

02114

Massachusetts Eye and Ear Infirmary, Boston

EC1V 2PD

Moorfields Eye Hospital, London