Study Summary
The goal of this clinical trial is to assess the efficacy and safety of GS010 gene therapy - (lenadogene nolparvovec) in subjects with LHON due to the G11778A ND4 mitochondrial mutation with a vision loss 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 was administrated via intravitreal injection containing 1.2/1.3E11 vg in 90 μL balanced sterile saline solution (BSSS).
PlaceboDRUG
The placebo is a balanced sterile saline solution (BSSS) used for IVT. The placebo was 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 |
| Department of Ophthalmology, Icahn School of Medicine at Mount Sinai | New York | New York | United States |
| Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University | Philadelphia | Pennsylvania | United States |
| Vanderbilt Eye Institute | Nashville | Tennessee | United States |
| Universitair Ziekenhuis Gent | Ghent | Belgium | |
| CHNO Les Quinze Vingts | Paris | Paris | France |
| IRCCS Istituto delle Scienze Neurologiche di Bologna (ISNB) 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 |