RESCUE study
Research type
Research Study
Full title
A Randomized, Double-Masked, Sham-Controlled, Pivotal Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 (rAAV2/2-ND4) in Subjects Affected for 6 Months or Less by Leber Hereditary Optic Neuropathy Due to the G11778A Mutation in the Mitochondrial NADH Dehydrogenase 4 Gene
IRAS ID
180772
Contact name
Patrick Yu Wai Man
Contact email
Sponsor organisation
GenSight Biologics
Eudract number
2015-001265-11
Duration of Study in the UK
2 years, 9 months, 0 days
Research summary
Leber hereditary optic neuropathy (LHON) is an inherited form of blindness caused by pathogenic mutations (spelling mistakes) within the genetic code contained within mitochondria. Mitochondria are the powerhouses within cells which produce most of the energy required for normal function. The majority of patients with LHON will carry a specific mutation within the mitochondrial ND4 gene (m.11778G>A).
This study will investigate the efficacy of a single injection into the eye of the study medication GS010 in treating patients affected with LHON who have had vision loss for 6 months or less and who carry a specific gene alteration within their mitochondrial DNA.
Before patients can enrol into the study, they must have been confirmed to carry the mitochondrial ND4 gene mutation (m.11778G>A) and fulfil further inclusion criteria. Screening may take up to 28 days.
Participation may last up to 96 weeks (1 year 11 months).
Approximately 40 participants above the age of 18 years will take part in this study in the UK, Germany, Italy, France and the United States.
Enrolled participants will be randomly allocated to receive either GS010 or sham (fake) treatment in a 1:1 allocation ratio to the right eye. The left eye will receive the treatment not allocated to the right eye. Therefore each participant will receive GS010 in one eye and sham treatment in the other eye.
Following the single injection into each eye (treatment phase), participants will return to the clinic periodically to have their health checked.REC name
London - West London & GTAC Research Ethics Committee
REC reference
15/LO/1308
Date of REC Opinion
30 Sep 2015
REC opinion
Further Information Favourable Opinion