EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005

Summary

The purpose of this clinical study was 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 (AMD).

Timeline

Start

2019-04-26

Primary completion

2024-04-05

Completion

2024-04-05

Outcome

Missed primary endpoint

Stopped: “Terminated for interim analysis demonstrating futility (trial highly unlikely to meet efficacy outcome). The trial is not ending early because of medical problems or concerns.”

registry analysis (superiority test); GT005 Low Dose [2E10 vg] vs Untreated Control; LS Mean Difference 0.976 (90% CI 0.150 to 1.803); mixed model repeated measures NCT04437368 ↗

registry analysis (superiority test); GT005 High Dose [2E11 vg] vs Untreated Control; LS Mean Difference 1.233 (90% CI 0.445 to 2.022); mixed model repeated measures NCT04437368 ↗

Publications

• Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.

Drugs

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

Indications