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

• Status: Not yet recruiting
• Trial end date: 2024-10-01
• Target enrollment: 0
• Participant gender: All

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 4 weeks, a 104-week masked Treatment Period, followed by an Open-label Extension (OLE) Period starting at Week 104 and lasting for up to 1 year. This study will have 4 treatments arms: 100 milligrams (mg) twice daily (bid), 200 mg bid, 400 mg once daily (qd), and matching placebo. Safety will be monitored throughout the study.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals

Criteria

Key Inclusion Criteria:

• Presentation of GA secondary to AMD in at least 1 eye.

• Study eye must have the specified visual acuity (range of 84 to 4 letters; 20/20 to 20/800) using Early Treatment Diabetic Retinopathy Study charts at a starting distance of 4 meters.

• GA area of 0.5 to 17.76 square millimeter (~0.25 to 7 disc area) per eye measured by FAF.

Key Exclusion Criteria:

• GA in either eye due to cause other than AMD.

• Have previously received intravitreal anti-vascular endothelial growth factor injections in study eye.

• Have previously received any complement/stem cell/gene therapy for any ophthalmological condition.

• Previous participation in interventional clinical studies for treatment of drusen, nascent GA or GA (except vitamins or minerals) irrespective of route of administration (ocular or systemic) in either eye.

• Presence of active ocular diseases in either eye that in the opinion of the Investigator compromises or confounds visual function or interferes with study assessments.

• Known or suspected complement deficiency.

• History or presence of any clinically relevant co-morbidities or any uncontrolled conditions.

• Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan) or any of its excipients.

Note: Additional inclusion/exclusion criteria may apply, per protocol.