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

Phase 1
Conditions
Geographic Atrophy (AD) secondary to Age-related Macular Degeneration (AMD).
MedDRA version: 20.1Level: LLTClassification code 10063947Term: Geographic atrophySystem Organ Class: 100000004853
Therapeutic area: Diseases [C] - Eye Diseases [C11]
Registration Number
EUCTR2021-001198-22-IT
Lead Sponsor
ALEXION PHARMACEUTICALS INCORPORATED
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Authorised-recruitment may be ongoing or finished
Sex
All
Target Recruitment
330
Inclusion Criteria

Key Inclusion Criteria:
• Age >= 70 years, male or female
• Presentation of GA secondary to AMD in at least 1 eye.
• Study eye must have the specified VA (range of 84 to 4 letters; 20/20 to 20/800) using Early Treatment Diabetic Retinopathy Study charts at starting distance of 4 meters.
• GA area of 0.5 to 17.76 mm2 (~0.25 to 7 disc area [DA]) per eye measured by FAF
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 330

Exclusion Criteria

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.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD.;Secondary Objective: To evaluate the effect of danicopan on disease progression utilizing anatomical measures compared to placebo<br>To evaluate the effect of danicopan on disease progression utilizing functional measures compared to placebo<br>To evaluate the PK and PD of danicopan in patients with GA secondary to AMD<br>To evaluate the safety and tolerability of danicopan in patients with GA secondary to AMD;Primary end point(s): Mean rate of change from Baseline at Week 52 in the square root (sqrt) of total GA lesion area (mm/year) in the study eye as measured by fundus autofluorescence (FAF);Timepoint(s) of evaluation of this end point: week 52
Secondary Outcome Measures
NameTimeMethod
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