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

Study Identifier:
ALXN2040-GA-201
ClinicalTrials.gov Identifier:
NCT05019521
EudraCT Identifier:
2021-001198-22
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: Danicopan
  • Drug: Placebo
Date
Aug 2021 - Jul 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 60+ years
Requirements Information
Inclusion and Exclusion Criteria
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
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.
  • 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.
Sex
Female & Male
Age
60+ years

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: Danicopan
  • Drug: Placebo
Date
Aug 2021 - Jul 2024
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 60+ years years
Requirements Information
Inclusion and Exclusion Criteria
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
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.
  • 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.

Protocol 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.

Trial Locations

Location
Status
Location
Research Site
Phoenix, Arizona, United States, 85020
Status
N/A
Location
Research Site
Beverly Hills, California, United States, 90211
Status
N/A
Location
Research Site
Encino, California, United States, 91436
Status
N/A
Location
Research Site
Fresno, California, United States, 93720
Status
N/A
Location
Research Site
Fullerton, California, United States, 92835
Status
N/A
Location
Research Site
La Jolla, California, United States, 92093
Status
N/A
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