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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)

Status
Completed
Phases
Phase 2
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-508571-37-00
Acronym
ALXN2040-GA-201
Enrollment
153
Registered
2024-05-31
Start date
2022-04-29
Completion date
2024-10-31
Last updated
2024-06-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Geographic atrophy (GA) secondary to Age-related Macular Degeneration (AMD)

Brief summary

Change from Baseline to Week 52 in the square root (sqrt) of total GA lesion area (mm) in the study eye as measured by fundus autofluorescence (FAF)

Detailed description

Change from Baseline to Week 104 in the sqrt of the total GA lesion area in the study eye as measured by FAF [Time Frame: Baseline, Week 52 and Week 104], Change from Baseline to Week 52 and Week 104 in the total GA lesion area in the study eye as measured by FAF [Time Frame: Baseline, Week 52 and Week 104], Change from Baseline to Week 52 and Week 104 in macular ellipsoid zone (EZ) and outer retinal integrity in the study eye, the fellow eye, and both eyes combined as measured by SD-OCT, Change from Baseline to Week 52 and Week 104 in subretinal pigment epithelium (sub-RPE) compartment/drusen/RPE complex in the study eye, fellow eye and both eyes combined as measured by SD-OCT., Change from Baseline to Week 52 and Week 104 in monocular Best corrected Visual Acuity (BCVA) scores in the study eye as assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart [Time Frame: Baseline, Week 52 and Week 104], Change from Baseline to Week 52 and Week 104 in monocular Low Luminance Visual Acuity (LLVA) scores in the study eye as assessed by the ETDRS chart [Time Frame: Baseline, Week 52 and Week 104], Change from Baseline to Week 52 and Week 104 in monocular low luminance deficit (BCVA-LLVA) in the study eye [Time Frame: Baseline, Week 52 and Week 104], Change form Baseline to Week 52 and Week 104 in monocular reading speeds in the study eye as assessed by Minnesota Low Vision Reading Test (MNRead) Acuity Charts or Radner Reading Chart [Time Frame: Baseline, Week 52 and Week 104], Change from Baseline to Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores [Time Frame: Baseline, Week 52 and Week 104], Plasma concentration of Danicopan over time [Time Frame: Up to 4 hours postdose], PD Biomarkers, Ex Vivo Serum Alternative Pathway Activity, and Plasma Bb Concentration Over Time [Time Frame: Up to 4 hours postdose], Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Ocular TEAEs, SAEs, and clinical laboratory abnormalities, and events leading to discontinuation of study drug throughout the study [Time Frame: Day 1 through Week 104]

Interventions

DRUGPlacebo matching ALXN2040

Sponsors

Alexion Pharmaceuticals Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
18 Years to No maximum

Design outcomes

Primary

MeasureTime frame
Change from Baseline to Week 52 in the square root (sqrt) of total GA lesion area (mm) in the study eye as measured by fundus autofluorescence (FAF)

Secondary

MeasureTime frame
Change from Baseline to Week 104 in the sqrt of the total GA lesion area in the study eye as measured by FAF [Time Frame: Baseline, Week 52 and Week 104], Change from Baseline to Week 52 and Week 104 in the total GA lesion area in the study eye as measured by FAF [Time Frame: Baseline, Week 52 and Week 104], Change from Baseline to Week 52 and Week 104 in macular ellipsoid zone (EZ) and outer retinal integrity in the study eye, the fellow eye, and both eyes combined as measured by SD-OCT, Change from Baseline to Week 52 and Week 104 in subretinal pigment epithelium (sub-RPE) compartment/drusen/RPE complex in the study eye, fellow eye and both eyes combined as measured by SD-OCT., Change from Baseline to Week 52 and Week 104 in monocular Best corrected Visual Acuity (BCVA) scores in the study eye as assessed by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart [Time Frame: Baseline, Week 52 and Week 104], Change from Baseline to Week 52 and Week 104 in monocular Low Luminanc

Countries

Czechia, France, Germany, Hungary, Italy, Latvia, Slovakia, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026