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

Sponsor organisation: Gyroscope Therapeutics Limited

Eudract number: 2017-003712-39

Duration of Study in the UK: 6 years, 7 months, 1 days

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.

Animation showing progression of atrophy over a 6 year time period.

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