EXPLORE
Research type
Research Study
Full title
EXPLORE: A Phase 2, outcomes assessor-masked, multicentre, randomised study to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with geographic atrophy secondary to age-related macular degeneration.
IRAS ID
273034
Contact name
Minal Kara
Contact email
Sponsor organisation
Gyroscope Therapeutics
Eudract number
2019-003421-22
Duration of Study in the UK
0 years, 11 months, 2 days
Research summary
Research Summary:
AMD is a disease of the eye caused when part of the retina deteriorates. The retina is the interior layer of the eye. The retina’s central portion, known as the macula, records the images we see and it controls our ability to read, drive a car, recognise faces or colours, and see objects in fine detail. AMD leads to progressive loss of vision. Geographic atrophy (GA) is when AMD progresses and causes further loss of vision or blurred vision.
The study aims to learn about the effects of the study drug(GT005). GT005 is a gene therapy that uses a genetically modified virus to make the cells in the eye produce more of a type of protein called Complement Factor I (CFI). Low levels of CFI are linked with AMD and GA. By raising the levels of CFI in the eye, it may be possible to reduce the progression of AMD and GA and so GT005 may help the disease. The study will also measure the safety of GT005 and patients with GA due to AMD.
Participants will be treated with either one of two doses of GT005 (2E10 vg and 2E11 vg) or no dose at all. If participants receive GT005 this will be given as a single injection into one eye. Participants assigned to no dose will not receive an injection.
This is a randomised, outcomes assessor-masked, study meaning neither the participant, nor the study doctor will know which dose of GT005 was given.
Gyroscope Therapeutics are sponsoring this multicentre study and it is anticipated that approximately 54 participants will be randomised to GT005 or uncontrolled treatment in a 2:1 ratio across the world.
Summary of results:
In September 2023, the sponsor decided to end the trial early because data analysis of this and other trials showed GT005 did not affect Geographical Atrophy (GA) more than no treatment. The decision was not due to safety concerns.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
19/SC/0540
Date of REC Opinion
23 Jan 2020
REC opinion
Further Information Favourable Opinion