GS-LHON-CLIN-05 REFLECT
Research type
Research Study
Full 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
IRAS ID
229343
Contact name
Patrick Yu Wai Man
Contact email
Sponsor organisation
GenSight Biologics
Eudract number
2017-002187-40
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 4 months, 9 days
Research summary
This is a phase III, multicentred randomised placebo controlled study in participants with Leber Hereditary Optic Neuropathy (LHON). Leber Hereditary Optic Neuropathy is due to a defect (mutation) in a mitochondrial gene that a person is born with causing a breakdown of the nerve that delivers messages from the eye. Genes are pieces of deoxyribonucleic acid (DNA) that hold genetic information about a person and they determine, for example, the colour of a persons eyes, height and skin colour. A defect in a gene can lead to a malfunction of cells and/or organs. Mitochondria are small structures contained in most cells which are responsible for the production of a cell’s energy and they contain their own DNA.
The purpose of the study is to evaluate the safety of treating eyes with GS010 and assess if GS010 is able to improve vision or prevent further vision loss caused by LHON. The study treatment GS010 is a gene therapy product, which is an experimental technique that uses genes to treat and prevent diseases. A gene is a region of DNA which are the instructions for growth and development of all living things.
The study is designed so that all participants will receive the experimental treatment into at least one eye. Participants may also receive placebo (a solution containing no study treatment) injection in the other eye. This will be assigned at random and neither the study doctor nor participant will know which eye received the active treatment.
The study will involve participants age 15 and older with LHON that meet the study entry requirement. The study is divided into four main visits: Screening Visit, Inclusion Visit, Treatment Visit and Post Treatment Visit. The participant will remain in follow up until two years after the initial injection.REC name
London - West London & GTAC Research Ethics Committee
REC reference
18/LO/0013
Date of REC Opinion
30 Jan 2018
REC opinion
Unfavourable Opinion