FOCUS: A study to test a new treatment for dry AMD
Research type
Research Study
Full title
FOCUS: AN OPEN LABEL FIRST IN HUMAN PHASE I/II MULTICENTRE STUDY TO EVALUATE THE SAFETY, DOSE RESPONSE AND EFFICACY OF GT005 ADMINISTERED AS A SINGLE SUBRETINAL INJECTION IN SUBJECTS WITH MACULAR ATROPHY DUE TO AGE-RELATED MACULAR DEGENERATION
IRAS ID
191852
Contact name
Steel
Contact email
Sponsor organisation
Medpace UK
Eudract number
2017-003712-39
Duration of Study in the UK
6 years, 7 months, 1 days
Research summary
Research Summary:
Age-related Macular Degeneration (AMD) is the most common cause of blindness among the elderly in the industrialised world- affecting more than 35 million people globally. AMD presents as a progressive loss of vision in the centre of the visual field resulting in a blurred area or blank spot in the centre of vision. With ageing, AMD progresses to an advanced stage becoming increasingly difficult for patients to recognise faces, drive, read and/or perform other activities of daily life. The overall UK prevalence of AMD is 2.4%, with a sharp age related increase in occurrence of 4.8% of people over 65 and as many as 12.2% over 80 years of age.
This is an open label first in human phase 1/2 study recruiting patients with confirmed AMD. The overall objective is to evaluate the safety, dose response and efficacy of a gene therapy (GT005) in AMD patients. The study will be conducted in three parts; Part 1: dose-escalation, Part 2: dose expansion, and Part 3 dose-escalation with Orbit Subretinal Delivery System(SDS). Parts 1 and 2 will deliver GT005 via the standard transvitreal procedure and Part 3 will deliver GT005 via the Orbit SDS device.
The study will be conducted at approximately 10 study sites; surgical procedures will be carried out at one or more central surgical site where the treatment is administered as a single subretinal injection into one eye.
Total study length is estimated to be 5 years, including a subject recruitment period estimated at 18 months and a 4 year post-treatment follow-up period.
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
18/SC/0016
Date of REC Opinion
20 Apr 2018
REC opinion
Further Information Favourable Opinion