NCT03846193

FOCUS: A Phase I/II First in Human Study to Evaluate the Safety and Efficacy of GT005 Administered in Subjects With Dry AMD

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 AMD

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 56 (actual)
• Sponsor: Gyroscope Therapeutics Limited · Industry
• Sex: All
• Age: 55 Years
• Healthy volunteers: Not accepted

Summary

This was an open label first in human Phase I/II multicentre study of GT005 in subjects with Macular Atrophy due to Age-related macular degeneration (AMD).

Detailed description

This was an open label first-in-human Phase I/II multicenter study to evaluate the safety, dose response and efficacy of GT005 in participants with Geographic atrophy (GA) due to Age-related macular degeneration (AMD). The study treatment GT005 consists of an Adeno-associated virus serotype 2 (AAV2) expressing human complement factor I (hCFI). The treatment was administered as a single subretinal administration in one eye - the "study eye". Both eyes were assessed at the screening visit. If both eyes meet the eligibility criteria, the study eye will be the worse seeing eye, or the eye with the largest geographic atrophy (GA) lesion area for eyes with equivalent visual acuity, unless the participant (in consultation with the surgeon) expresses an alternative preference. The study was conducted in 4 parts: in Part 1 and Part 2 GT005 was administered subretinally via transvitreal procedure; in Part 3 and 4 GT005 was delivered subretinally via a suprachoroidal cannulation with the Orbit Subretinal delivery system (SDS). The Orbit SDS is a 510(k) cleared device in the US, whereby Parts 3 and 4 were only conducted at US sites. The treatment consisted in 3 dose levels: low dose, 2E10 vector genomes (vg); medium dose, 5E10 vg; and high dose, 2E11 vg. The study consisted of up to 13 visits over a 5-year period. All participants were assessed for the occurrence of treatment emergent adverse events (AE)s at each visit and underwent visual function and retinal imaging assessments and biological sampling as per the schedule of assessments. This study was conducted in compliance with Independent ethics committees (IECs) / Institutional review boards (IRBs), informed consent regulations, the Declaration of Helsinki, nternational Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) Guidelines, and the Food and Drug Administration (FDA) guidance.

Conditions

• Dry Age-related Macular Degeneration
• Macular Degeneration
• Retinal Disease
• Eye Diseases
• Retinal Degeneration
• Geographic Atrophy
• Macular Atrophy

Interventions

Type	Name	Description
BIOLOGICAL	GT005	GT005 is a recombinant, non-replicating AAV2 expressing human complement factor I (CFI). GT005 was administered as a single time subretinal injection into the study eye of subjects allocated to one of the two GT005 doses.
DEVICE	GT005 / Device: Orbit™ Subretinal Delivery System	GT005 is a recombinant, non-replicating AAV2 expressing human complement factor I (CFI). A single dose of GT005 was administered with subretinal injection via suprachoroidal cannulation approach. Device: Orbit™ Subretinal Delivery System

Timeline

• Start date: 2018-12-17
• Primary completion: 2024-06-25
• Completion: 2024-06-25
• First posted: 2019-02-19
• Last updated: 2026-01-28
• Results posted: 2025-08-24

Locations

13 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study
• FDA-regulated device study