Proof of concept and dose-finding study of danicopan in patients with GA secondary to AMD
Research type
Research Study
Full title
A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study of Danicopan (ALXN2040) in Patients with Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
IRAS ID
1004135
Contact name
Aleks Skuban
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2021-001198-22
Clinicaltrials.gov Identifier
Research summary
Age-related macular degeneration (AMD) is a common condition that affects the central vision. It usually affects people aged 50 years or over. Geographic atrophy (GA) is an advanced form of AMD and there is currently no treatment. The purpose of this double-masked study is to determine whether treatment with Danicopan (an oral complement alternative pathway factor D inhibitor) is effective in treating adults with GA secondary to AMD. Danicopan is an investigational drug being developed by Alexion Pharmaceuticals, Inc..
About 330 adults at least 70 years of age will take part in this study. This study will be conducted at approximately 123 sites in approximately 15 countries globally. Participants will be randomized (1:1:1:1) to one of four treatment groups (3 active treatment groups and 1 placebo group): 100 mg twice daily (bid) dose group, 200 mg bid dose group, 400 mg once daily (qd) dose group, and a placebo group.
The study has the following periods: screening period (up to 4 weeks), a masked treatment period (Years 1 and 2), and an open-label extension (OLE) period (Year 3). Masked treatment means neither the participant nor treating doctor knows if treatment is with active drug or placebo; open-label means the participant and doctor both know. In addition, there is a final follow-up visit 30 days (+ 7 days) after receiving the last dose. Participants will be in the study for up to 166 weeks (about 3 years and 2 months).
REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
21/LO/0792
Date of REC Opinion
11 Feb 2022
REC opinion
Further Information Favourable Opinion