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

Alexion Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Geographic Atrophy

Treatments: Drug: Danicopan; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05019521

ALXN2040-GA-201

2021-001198-22 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 365 patients

Sex: All

Ages: 60+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.
• 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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365 participants in 4 patient groups, including a placebo group

Danicopan: 100 mg

Experimental group

Description:

Participants will receive danicopan 100 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Treatment:

Drug: Danicopan

Danicopan: 200 mg

Experimental group

Description:

Participants will receive danicopan 200 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Treatment:

Drug: Danicopan

Danicopan: 400 mg

Experimental group

Description:

Participants will receive danicopan 400 mg qd during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Treatment:

Drug: Danicopan

Placebo

Placebo Comparator group

Description:

Participants will receive matching placebo and will be re-randomized to one of the active treatment groups at Week 52, or to the optimal dose, if already identified.

Treatment:

Drug: Placebo

Drug: Danicopan