Study title:
Efficacy and safety of bilateral intravitreal injection of GS010: a randomized, double-masked, placebo-controlled trial in subjects affected with G11778A ND4 leber hereditary optic neuropathy for up to one year.
Long title:
Efficacy and safety of bilateral intravitreal injection of GS010: a randomized, double-masked, placebo-controlled trial in subjects affected with G11778A ND4 leber hereditary optic neuropathy for up to one year.
Date receipt dossier:
21 fév 2018
EudraCT number:
2017-002187-40
Pharmaceutical study code:
GS010
Company / Sponsor:
GenSight Biologics
Treated organism:
Humans
Indication category:
Genetic disorders
Disease:
Leber hereditary optic neuropathy
Therapeutic approach:
Gene therapy
Genetic modification:
human wild-type MT-NDA (mitochondrial NADH Dehydrogenase 4)
Method of transfer of nucleic acid of interest:
AAV2
Administered biological material:
Recombinant AAV2 expressing MT-NDA
Route of administration:
Intravitreal
Locations in Belgium:
Universitair Ziekenhuis Gent
Type of procedure:
Contained use only
Current status:
Authorized