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


Brief 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.

Primary Purpose
Study Type
Phase 2


Healthy Volunteers
Minimum Age
60 Years
Maximum Age

Key Inclusion Criteria:

  • Vaccination for Neisseria meningitidis.
  • Capable of giving signed informed consent.
  • Presentation of GA secondary to AMD in at least 1 eye
  • The entire GA lesion must be > 1 μm outside of the foveal center

Key Exclusion Criteria:

  • GA in the study eye due to cause other than AMD.
  • Have previously received intravitreal anti-vascular endothelial growth factor injections in study eye for intraocular vascular disease.
  • Have previously received any stem cell/gene therapy for any ophthalmological condition in either eye.
  • Previous participation in interventional clinical studies for any ophthalmic indications in the study eye regardless of route of administration within the last 3 months or 5 half-lives of the investigational product (whichever is longer).
  • Presence of active ocular diseases in the study 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 medical or psychological condition that, in the opinion of the Principal Investigator, would make the patient inappropriate for the study.
  • Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan) or any of its excipients.

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

Join this Trial

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Study Stats
Protocol No.
Eye/Ocular Disorders
Melissa Sedaghat
  • UCLA Westwood
For Providers
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