Study Summary
The goal of this clinical trial is to assess the safety and efficacy of GS010, a gene therapy, in improving the retina functional \& structural outcomes in subjects with LHON due to the G11778A ND4 mitochondrial mutation when vision loss duration is present up to one year.
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GS010GENETIC
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.
PlaceboDRUG
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.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Doheny Eye Center UCLA Pasadena | Pasadena | California | United States |
| University of Colorado Health Eye Center | Aurora | Colorado | United States |
| Emory Healthcare - The Emory Clinic | Atlanta | Georgia | United States |
| Massachusetts Eye and Ear Infirmary | Boston | Massachusetts | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | United States |
| Wills Eye Institute - Ocular Oncology Service | Philadelphia | Pennsylvania | United States |
| Vanderbilt Eye Institute | Nashville | Tennessee | United States |
| Universitair Ziekenhuis Gent | Ghent | Belgium | |
| CHNO Les Quinze Vingts | Paris | France | |
| IRCCS Istituto delle Scienze Neurologiche di Bologna UOC Clinica Neurologica | Bologna | Italy | |
| Hospital Universitario Ramon y Cajal | Madrid | Spain | |
| Taipei Veterans General Hospital | Taipei | Taiwan | |
| Moorfields Eye Hospital | London | Greater London | United Kingdom |