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

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)

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.

Study Overview

• Status: Terminated
• Conditions: Geographic Atrophy
• Intervention / Treatment: Drug: Danicopan; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 365
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • Adelaide, Australia, 5000
    • Research Site
  • Albury, Australia, 2640
    • Research Site
  • East Melbourne, Australia, 3002
    • Research Site
  • Hurstville, Australia, 2220
    • Research Site
  • Parramatta, Australia, 2150
    • Research Site
  • Strathfield, Australia, 2135
    • Research Site
  • Sydney, Australia, 2000
    • Research Site
• Czechia
  • Pardubice, Czechia, 530 02
    • Research Site
  • Prague, Czechia, 150 00
    • Research Site
• France
  • Créteil, France, 94010
    • Research Site
  • Lyon, France, 69004
    • Research Site
  • Marseille, France, 13008
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  • Nantes, France, 44000
    • Research Site
  • Nice, France, 06000
    • Research Site
  • Paris, France, 75010
    • Research Site
  • Paris, France, 75019
    • Research Site
  • Paris, France, 75571
    • Research Site
  • Poitiers, France, 86021
    • Research Site
  • Strasbourg, France, 67091
    • Research Site
  • Écully, France, 69130
    • Research Site
• Germany
  • Berlin, Germany, 12203
    • Research Site
  • Bonn, Germany, 53127
    • Research Site
  • Düsseldorf, Germany, 40225
    • Research Site
  • Freiburg im Breisgau, Germany, 79106
    • Research Site
  • Göttingen, Germany, 37075
    • Research Site
  • Hanover, Germany, 30625
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  • Heidelberg, Germany, 69120
    • Research Site
  • Mainz A. Rhein, Germany, 55131
    • Research Site
  • Marburg, Germany, 35043
    • Research Site
  • München, Germany, 81675
    • Research Site
  • Münster, Germany, 48149
    • Research Site
  • Münster, Germany, 48145
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  • Neubrandenburg, Germany, 17036
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  • Regensburg, Germany, 93053
    • Research Site
  • Tübingen, Germany, 72076
    • Research Site
  • Ulm, Germany, 89081
    • Research Site
  • Würzburg, Germany, 97080
    • Research Site
• Hungary
  • Budapest, Hungary, 1133
    • Research Site
  • Pécs, Hungary, 7621
    • Research Site
• Italy
  • Florence, Italy, 50134
    • Research Site
  • Genova, Italy, 16132
    • Research Site
  • Milan, Italy, 20122
    • Research Site
  • Milan, Italy, 20132
    • Research Site
  • Milan, Italy, 20157
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  • Naples, Italy, 80131
    • Research Site
  • Roma, Italy, 00168
    • Research Site
  • Roma, Italy, 00133
    • Research Site
• Japan
  • Fukushima, Japan, 960-1295
    • Research Site
  • Kita-gun, Japan, 761-0793
    • Research Site
  • Osaka, Japan, 5458586
    • Research Site
• Latvia
  • Riga, Latvia, LV1002
    • Research Site
  • Riga, Latvia, LV-1009
    • Research Site
• Slovakia
  • Bratislava, Slovakia, 811 08
    • Research Site
  • Žilina, Slovakia, 012 07
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• South Korea
  • Busan, South Korea, 49241
    • Research Site
  • Daegu, South Korea, 42415
    • Research Site
  • Incheon, South Korea, 22332
    • Research Site
  • Jinju, South Korea, 52727
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  • Seoul, South Korea, 06351
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  • Seoul, South Korea, 03082
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• Spain
  • Barcelona, Spain, 08035
    • Research Site
  • Barcelona, Spain, 8025
    • Research Site
  • Barcelona, Spain, 08022
    • Research Site
  • Bilbao, Spain, 48006
    • Research Site
  • Córdoba, Spain, 14012
    • Research Site
  • Majadahonda, Spain, 28222
    • Research Site
  • Pamplona, Spain, 31005
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  • Seville, Spain, 41071
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  • Valencia, Spain, 46014
    • Research Site
  • Valladolid, Spain, 46012
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  • Zaragoza, Spain, 50009
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• United Kingdom
  • Bristol, United Kingdom, BS1 2LX
    • Research Site
  • Liverpool, United Kingdom, L69 3BX
    • Research Site
  • London, United Kingdom, SE5 9RS
    • Research Site
  • London, United Kingdom, EC1V 2PD
    • Research Site
  • London, United Kingdom, GU16 7UJ
    • Research Site
  • London, United Kingdom, NW1 5QH
    • Research Site
  • Newcastle upon Tyne, United Kingdom, NE7 7DN
    • Research Site
  • Southampton, United Kingdom, S016 6YD
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• United States
  • Arizona
    • Phoenix, Arizona, United States, 85020
      • Research Site
  • California
    • Beverly Hills, California, United States, 90211
      • Research Site
    • Encino, California, United States, 91436
      • Research Site
    • Fresno, California, United States, 93720
      • Research Site
    • Fullerton, California, United States, 92835
      • Research Site
    • La Jolla, California, United States, 92093
      • Research Site
    • Pasadena, California, United States, 91105
      • Research Site
    • Sacramento, California, United States, 95841
      • Research Site
    • San Mateo, California, United States, 94401
      • Research Site
    • Walnut Creek, California, United States, 94598
      • Research Site
  • Colorado
    • Colorado Springs, Colorado, United States, 80909
      • Research Site
    • Longmont, Colorado, United States, 80503
      • Research Site
  • Connecticut
    • Waterford, Connecticut, United States, 06385
      • Research Site
  • Florida
    • Clearwater, Florida, United States, 33761
      • Research Site
    • Coral Springs, Florida, United States, 33067
      • Research Site
    • Deerfield Beach, Florida, United States, 33064
      • Research Site
    • Naples, Florida, United States, 34103
      • Research Site
    • Palm Beach Gardens, Florida, United States, 33418
      • Research Site
    • St. Petersburg, Florida, United States, 33711
      • Research Site
  • Georgia
    • Augusta, Georgia, United States, 30909
      • Research Site
  • Indiana
    • New Albany, Indiana, United States, 47150
      • Research Site
  • Iowa
    • West Des Moines, Iowa, United States, 50266
      • Research Site
  • Maryland
    • Hagerstown, Maryland, United States, 21740
      • Research Site
    • Rosedale, Maryland, United States, 21237
      • Research Site
    • Towson, Maryland, United States, 21204
      • Research Site
  • Massachusetts
    • Springfield, Massachusetts, United States, 01107
      • Research Site
  • Mississippi
    • Southaven, Mississippi, United States, 38671
      • Research Site
  • Nevada
    • Reno, Nevada, United States, 89502
      • Research Site
  • New Jersey
    • Teaneck, New Jersey, United States, 07666
      • Research Site
  • New York
    • Great Neck, New York, United States, 11021
      • Research Site
  • North Carolina
    • Durham, North Carolina, United States, 27705
      • Research Site
  • Ohio
    • Cincinnati, Ohio, United States, 45219
      • Research Site
  • Oregon
    • Portland, Oregon, United States, 97225
      • Research Site
    • Springfield, Oregon, United States, 97477
      • Research Site
  • Pennsylvania
    • Kingston, Pennsylvania, United States, 18704
      • Research Site
    • Monroeville, Pennsylvania, United States, 15146
      • Research Site
    • Philadelphia, Pennsylvania, United States, 19107
      • Research Site
    • Philadelphia, Pennsylvania, United States, 19104-2640
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  • South Carolina
    • Greenville, South Carolina, United States, 29605
      • Research Site
    • Ladson, South Carolina, United States, 29456
      • Research Site
  • South Dakota
    • Rapid City, South Dakota, United States, 57701
      • Research Site
  • Texas
    • Austin, Texas, United States, 78750
      • Research Site
    • Bellaire, Texas, United States, 77401
      • Research Site
    • Conroe, Texas, United States, 77384
      • Research Site
    • Dallas, Texas, United States, 75231
      • Research Site
    • San Antonio, Texas, United States, 78240
      • Research Site
    • Southlake, Texas, United States, 76092
      • Research Site
  • Virginia
    • Fairfax, Virginia, United States, 22031
      • Research Site
    • Lynchburg, Virginia, United States, 24502
      • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 60 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Danicopan: 100 mg; 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.	Drug: Danicopan; Oral tablet.; Other Names: ALXN2040
Experimental: Danicopan: 200 mg; 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.	Drug: Danicopan; Oral tablet.; Other Names: ALXN2040
Experimental: Danicopan: 400 mg; 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.	Drug: Danicopan; Oral tablet.; Other Names: ALXN2040
Placebo Comparator: Placebo; 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.	Drug: Danicopan; Oral tablet.; Other Names: ALXN2040; Drug: Placebo; Oral tablet.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF)	FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area measurement. The total GA lesion area (square millimeter [mm^2]) was transformed into sqrt (millimeter [mm]). Greater area affected means a worse outcome than smaller area affected. The rate of change from Baseline (mm/year) for each treatment group was estimated based on the coefficients for time and the interaction of time and treatment from the mixed models for repeated measures (MMRM). MMRM analysis included the change from Baseline at post-baseline visit in the sqrt GA lesion area as dependent variable.	Baseline, Week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF	FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area measurement. The total GA lesion area (mm^2) was transformed into sqrt (mm). Greater area affected means a worse outcome than smaller area affected.	Baseline, 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	FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area (mm^2) measurement. Greater area affected means a worse outcome than smaller area affected.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT)	Photoreceptor loss was assessed by measuring the percent of macular EZ total attenuation using SD-OCT. The EZ was considered to be formed mainly by mitochondria within the ellipsoid layer of the outer portion of the inner segments of the photoreceptors. In SD-OCT, decreases in the integrity and intensity of the EZ have been correlated with the reduction in cone photoreceptors and retinal sensitivity in retinal degenerative diseases. Lower increase in percentage means a better outcome. Higher increase in percentage means a worse outcome.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	Photoreceptor degradation was assessed by measuring the percent of macular EZ partial attenuation using SD-OCT. The EZ was considered to be formed mainly by mitochondria within the ellipsoid layer of the outer portion of the inner segments of the photoreceptors. In SD-OCT, decreases in the integrity and intensity of the EZ have been correlated with the reduction in cone photoreceptors and retinal sensitivity in retinal degenerative diseases. Lower increase in percentage means a better outcome. Higher increase in percentage means a worse outcome.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	The ONL - RPE mean central 1 mm subfield thickness refers to the average thickness of the retinal layers between the ONL and RPE measured within a 1 mm radius from the foveal center (the central point of the retina).	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	The ONL - RPE mean central macular 2 mm subfield thickness refers to the thickness of the retina in the central part of the macula, specifically within a 2 mm radius.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	The EZ - RPE macular volume refers to the volume of the retinal tissue between the EZ and the RPE, specifically within the macular region. Changes in EZ-RPE volume indicate the presence or progression of diseases affecting the outer retina.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	The EZ - RPE mean central 1 mm subfield thickness is a measurement of the thickness of the ellipsoid zone, a layer within the retina, in the central 1 mm region. Changes in EZ-RPE thickness indicate damage or dysfunction within the photoreceptors, which is associated with various eye diseases like AMD.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	The EZ - RPE mean central macular 2 mm thickness measurement reflects the distance between the outer border of the EZ and the inner border of the RPE layer, typically within a central 2 mm area. Decreased EZ-RPE thickness is an early indicator of disease, such as in dry AMD, where a thinning or loss of the EZ is a hallmark. Changes in EZ-RPE thickness is used to monitor treatment response in conditions like AMD.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	The percentage of total attenuation of the EZ in the central 1 mm subfield is reported.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	The percentage of partial attenuation of the EZ in the central 1 mm subfield is reported.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	The percentage of total attenuation of the EZ in the central macular 2 mm subfield is reported.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT	The percentage of partial attenuation of the EZ in the central macular 2 mm subfield is reported.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Sub-RPE Central 1 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT	Sub-RPE compartment measures reflect the disease burden between the RPE and Bruch's membrane. The sub-RPE central 1 mm mean thickness refers to the average thickness of the retina within a 1-mm diameter circular area centered around the fovea (the central part of the retina).	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Sub-RPE Central Macular 2 mm Subfield Mean Thickness in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT	Sub-RPE compartment measures reflect the disease burden between the RPE and Bruch's membrane. The sub-RPE central macular 2 mm subfield thickness refers to the thickness of the retina within a 2-mm radius in the central part of the macula.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Panmacular Sub-RPE Volume in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT	Sub-RPE compartment measures reflect the disease burden between the RPE and Bruch's membrane. The panmacular sub-RPE volume refers to the total volume of fluid or space located underneath the RPE across the entire macula (the central part of the retina).	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Percent Macular Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT	Macular total RPE attenuation refers to the degree of thinning or loss of the RPE in the macula, the central part of the retina responsible for sharp, detailed vision. This was measured as a percentage of the macular area affected.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Percent Central 1 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT	The percentage of total attenuation of the RPE in the central 1 mm subfield is reported.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Percent Central Macular 2 mm Subfield Total RPE Attenuation in the Study Eye, Fellow Eye and Both Eyes Combined as Measured by SD-OCT	The percentage of total attenuation of the RPE in the central macular 2 mm subfield is reported.	Baseline, Week 52 and Week 104
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	ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome).	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	LLVA was measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the participant read the normally illuminated ETDRS chart. LLVA was reported as number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision (or VA). An increase in the number of letters read correctly means that vision has improved. Change from baseline: a negative score indicates decline in LLVA.	Baseline, Week 52 and Week 104
Change From Baseline to Week 52 and Week 104 in Low Luminance Deficit (LLD) (BCVA-LLVA) Score in the Study Eye	LLD score was calculated as the difference between BCVA and LLVA (BCVA-LLVA) scores. BCVA and LLVA were reported as the number of letters read correctly by the participant using the ETDRS Scale (ranging from 0 to 100 letters). The lower the number of letters read correctly on the eye chart, the worse the vision. LLD is a measure of visual acuity impairment in low-light conditions. A higher LLD score indicates a greater difference between vision in bright and dim light, suggesting a more significant loss of visual function in low-luminance settings.	Baseline, Week 52 and Week 104
Change From 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 Charts	MNRead acuity cards were continuous-text reading-acuity cards suitable for measuring the reading acuity and reading speed of normal and low-vision participants. The MNRead acuity cards consisted of single, simple sentences with equal numbers of characters. A stopwatch was used to record time to a tenth of a second. Sentences that could not be read or were not attempted due to vision should be recorded as 0 for time and 10 for errors. The Radner Reading Cards were suitable for measuring reading speed, reading visual acuity, and critical print size. The reading test was stopped when the reading time was longer than 20 seconds or when the participant was making severe errors. A negative change from baseline indicates a decrease in the monocular reading speed; disease worsening.	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) Composite Scores	The NEI VFQ-25 instrument measures dimensions of self-reported vision-targeted health status of individuals with chronic eye conditions. The NEI VFQ-25 consists of 11 vision related domains: global vision rating, difficulty with near vision activities, difficulty with distance vision activities, limitations in social function related to vision, role limitations, dependency on others due to vision, mental health symptoms due to vision, driving difficulties, limitations with peripheral- and color-vision, and ocular pain. The NEI VFQ-25 also includes a single item measuring general health. A composite score averages the vision related domains and ranges from 0 (worse) to 100 (best). A higher score represents better functioning. A negative change from baseline indicates a decrease in the visual functioning; disease worsening.	Baseline, Week 52 and Week 104
Plasma Concentration of Danicopan	All values below the limit of quantification (<0.1 nanograms [ng]/milliliter [mL]) were set to 0 for calculation of summary statistics.	Day 1 and Week 52
Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity	Serum AP functional activity was measured by the Wieslab functional immunoassay method.	Week 52 (pre-dose, 2 hours post-dose)
Change From Baseline in Plasma Bb Fragment of Complement Factor B (Bb) Concentration		Baseline, Week 52 (pre-dose)
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Events Leading To Discontinuation of Study Drug Throughout the Study	An adverse event (AE) was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, resulted in persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), or an important medical event or reaction. A TEAE was defined as any AE that started during or after the first dose of study intervention. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.	Day 1 through Week 108

Collaborators and Investigators

• Sponsor: Alexion Pharmaceuticals, Inc.

Publications and helpful links

General Publications

• Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): August 23, 2021
• Primary Completion (Actual): July 5, 2024
• Study Completion (Actual): January 21, 2025

Study Registration Dates

• First Submitted: August 19, 2021
• First Submitted That Met QC Criteria: August 19, 2021
• First Posted (Actual): August 25, 2021

Study Record Updates

• Last Update Posted (Estimated): September 5, 2025
• Last Update Submitted That Met QC Criteria: August 15, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: AMD; Danicopan; ALXN2040; Geographic Atrophy; GA; Complement Inhibition
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Geographic Atrophy; Substandard Drugs; Pharmaceutical Preparations; Counterfeit Drugs; danicopan
• Other Study ID Numbers: ALXN2040-GA-201; 2021-001198-22 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No