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

Phase 1/2 Terminated 56 enrolled Gyroscope Therapeutics Limited Novartis Pharmaceuticals · collab

Non-randomizedSequentialOpen-labelTreatment

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).

Timeline

Start: 2018-12-17
Primary completion: 2024-06-25
Completion: 2024-06-25

Outcome

Outcome not reported

Stopped: “The study was terminated due to the interim analysis demonstrating lack of treatment efficacy.”

Publications

Dreismann AK, McClements ME, Barnard AR, Orhan E, Hughes JP, Lachmann PJ, MacLaren RE. Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells. Gene Ther. 2021 May;28(5):265-276. doi: 10.1038/s41434-021-00239-9. Epub 2021 Mar 10.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	GT005	Gene therapy (AAV / viral vector)	2e+10 vg	Other
Subject	GT005	Gene therapy (AAV / viral vector)	5e+10 vg	Other
Subject	GT005	Gene therapy (AAV / viral vector)	2e+11 vg	Other