A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Summary

This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD. The study consists of a Screening Period of up to 6 weeks, a 104-week masked Treatment Period, followed by a 30-day Follow-up after the last dose. This study will have 4 treatments arms: 100 milligrams (mg) twice daily (bid), 200 mg bid, 400 mg once daily (qd), and matching placebo.

Timeline

Start

2021-08-23

Primary completion

2024-07-05

Completion

2025-01-21

Outcome

Missed primary endpoint

Stopped: “Lack of efficacy.”

registry analysis (superiority test); Danicopan 100 mg BID vs Placebo; p = 0.9205 NCT05019521 ↗

registry analysis (superiority test); Danicopan 200 mg BID vs Placebo; p = 0.9205 NCT05019521 ↗

registry analysis (superiority test); Danicopan 400 mg QD vs Placebo; p = 0.9205 NCT05019521 ↗

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	Danicopan	Small molecule	100 mg	Oral
Subject	Danicopan	Small molecule	200 mg	Oral
Subject	Danicopan	Small molecule	400 mg	Oral

Indications