Summary

Eligibility
for people ages 60 years and up (full criteria)
Location
at UCLA
Dates
study started
completion around

Description

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.

Official 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)

Keywords

Geographic Atrophy, GA, Danicopan, ALXN2040, Complement Inhibition, AMD, Macular Degeneration, Atrophy, Danicopan: 100 mg, Danicopan: 200 mg, Danicopan: 400 mg

Eligibility

You can join if…

Open to people ages 60 years and up

  • 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

You CAN'T join if...

  • 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.
  • Use of any investigational medicinal product (ie, participation in interventional clinical studies for any ophthalmic indications) or use of any regulatory approved treatment for GA in the study eye regardless of route of administration within the last 3 months or 5 half-lives of the last dose of the investigational or commercial 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.

Locations

  • Research Site
    Los Angeles California 90095 United States
  • Research Site
    Beverly Hills California 90211 United States
  • Research Site
    La Jolla California 92093 United States
  • Research Site
    Encino California 91436 United States
  • Research Site
    Fresno California 93720 United States
  • Research Site
    San Mateo California 94401 United States
  • Research Site
    Pasadena California 91105 United States
  • Research Site
    Fullerton California 92835 United States
  • Research Site
    Sacramento California 95841 United States
  • Research Site
    Walnut Creek California 94598 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Alexion Pharmaceuticals, Inc.
ID
NCT05019521
Phase
Phase 2 research study
Study Type
Interventional
Participants
About 365 people participating
Last Updated