A Study of Danicopan in Participants With Geographic Atro... | NCT05019521 | Trialant
NCT05019521
Sponsor
Alexion Pharmaceuticals, Inc.
Status
Terminated
Last Update Posted
Sep 5, 2025Actual
Enrollment
365Actual
Phase
Phase 2
Conditions
Geographic Atrophy
Interventions
Danicopan
Placebo
Countries
United States
Australia
Czechia
France
Germany
Hungary
Italy
Japan
Latvia
Slovakia
South Korea
Spain
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT05019521
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
ALXN2040-GA-201
Secondary IDs
ID
Type
Description
Link
2021-001198-22
EudraCT Number
Brief Title
A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
Official Title
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)
Acronym
Not provided
Organization
Alexion Pharmaceuticals, Inc.INDUSTRY
Status Module
Record Verification Date
Aug 2025
Overall Recruitment Status or Expanded Access Status
Terminated
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Lack of efficacy.
Expanded Access Info
No
Start Date
Aug 23, 2021Actual
Primary Completion Date
Jul 5, 2024Actual
Completion Date
Jan 21, 2025Actual
First Submitted Date
Aug 19, 2021
First Submission Date that Met QC Criteria
Aug 19, 2021
First Posted Date
Aug 25, 2021Actual
Results Waived
Not provided
Results First Submitted Date
Jul 2, 2025
Results First Submitted that Met QC Criteria
Aug 15, 2025
Results First Posted Date
Sep 5, 2025Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Aug 15, 2025
Last Update Posted Date
Sep 5, 2025Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Alexion Pharmaceuticals, Inc.INDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief 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.
Detailed Description
Not provided
Conditions Module
Conditions
Geographic Atrophy
Keywords
Geographic Atrophy
GA
Danicopan
ALXN2040
Complement Inhibition
AMD
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
365Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Danicopan: 100 mg
Experimental
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
Danicopan: 200 mg
Experimental
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
Danicopan: 400 mg
Experimental
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
Placebo
Placebo Comparator
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
Drug: Placebo
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Danicopan
Drug
Oral tablet.
Danicopan: 100 mg
Danicopan: 200 mg
Danicopan: 400 mg
Placebo
Outcomes Module
Primary Outcomes
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 Outcomes
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.
Other Outcomes
Not provided
Eligibility Module
Eligibility 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.
For standardization purposes, the primary and secondary efficacy analyses were measured in the pre-specified study eye per individual participant. In this study, ocular assessments were performed in both eyes at Screening. The eye that met all eligibility criteria was designated as the study eye and the other eye was used as the fellow eye. If both eyes were eligible, the right eye was designated as the study eye.
Recruitment Details
The study included 104-week Masked Treatment Period (Masked Placebo-controlled [Primary Evaluation Period; up to 52 weeks] and Masked Active Treatment [Secondary Evaluation Period; Week 52 to Week 104]). As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Danicopan 100 mg BID
Participants received danicopan 100 milligrams (mg) twice daily (BID) during the 104-week Masked Treatment Period.
FG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
Periods
Title
Milestones
Reasons Not Completed
Primary Evaluation Period (52 Weeks)
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
Oct 30, 2023
Jul 2, 2025
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Canada
Submission Tracking
Estimated Results First Submitted Date
Not provided
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
Quadruple
Masking Description
Masking of treatment allocation will be observed until 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
Beverly Hills
California
90211
United States
Research Site
Encino
California
91436
United States
Research Site
Fresno
California
93720
United States
Research Site
Fullerton
California
92835
United States
Research Site
La Jolla
California
92093
United States
Research Site
Pasadena
California
91105
United States
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Sacramento
California
95841
United States
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San Mateo
California
94401
United States
Research Site
Walnut Creek
California
94598
United States
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Colorado Springs
Colorado
80909
United States
Research Site
Longmont
Colorado
80503
United States
Research Site
Waterford
Connecticut
06385
United States
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Clearwater
Florida
33761
United States
Research Site
Coral Springs
Florida
33067
United States
Research Site
Deerfield Beach
Florida
33064
United States
Research Site
Naples
Florida
34103
United States
Research Site
Palm Beach Gardens
Florida
33418
United States
Research Site
St. Petersburg
Florida
33711
United States
Research Site
Augusta
Georgia
30909
United States
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New Albany
Indiana
47150
United States
Research Site
West Des Moines
Iowa
50266
United States
Research Site
Hagerstown
Maryland
21740
United States
Research Site
Rosedale
Maryland
21237
United States
Research Site
Towson
Maryland
21204
United States
Research Site
Springfield
Massachusetts
01107
United States
Research Site
Southaven
Mississippi
38671
United States
Research Site
Reno
Nevada
89502
United States
Research Site
Teaneck
New Jersey
07666
United States
Research Site
Great Neck
New York
11021
United States
Research Site
Durham
North Carolina
27705
United States
Research Site
Cincinnati
Ohio
45219
United States
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Portland
Oregon
97225
United States
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Springfield
Oregon
97477
United States
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Kingston
Pennsylvania
18704
United States
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Monroeville
Pennsylvania
15146
United States
Research Site
Philadelphia
Pennsylvania
19104-2640
United States
Research Site
Philadelphia
Pennsylvania
19107
United States
Research Site
Greenville
South Carolina
29605
United States
Research Site
Ladson
South Carolina
29456
United States
Research Site
Rapid City
South Dakota
57701
United States
Research Site
Austin
Texas
78750
United States
Research Site
Bellaire
Texas
77401
United States
Research Site
Conroe
Texas
77384
United States
Research Site
Dallas
Texas
75231
United States
Research Site
San Antonio
Texas
78240
United States
Research Site
Southlake
Texas
76092
United States
Research Site
Fairfax
Virginia
22031
United States
Research Site
Lynchburg
Virginia
24502
United States
Research Site
Adelaide
5000
Australia
Research Site
Albury
2640
Australia
Research Site
East Melbourne
3002
Australia
Research Site
Hurstville
2220
Australia
Research Site
Parramatta
2150
Australia
Research Site
Strathfield
2135
Australia
Research Site
Sydney
2000
Australia
Research Site
Pardubice
530 02
Czechia
Research Site
Prague
150 00
Czechia
Research Site
Créteil
94010
France
Research Site
Écully
69130
France
Research Site
Lyon
69004
France
Research Site
Marseille
13008
France
Research Site
Nantes
44000
France
Research Site
Nice
06000
France
Research Site
Paris
75010
France
Research Site
Paris
75019
France
Research Site
Paris
75571
France
Research Site
Poitiers
86021
France
Research Site
Strasbourg
67091
France
Research Site
Berlin
12203
Germany
Research Site
Bonn
53127
Germany
Research Site
Düsseldorf
40225
Germany
Research Site
Freiburg im Breisgau
79106
Germany
Research Site
Göttingen
37075
Germany
Research Site
Hanover
30625
Germany
Research Site
Heidelberg
69120
Germany
Research Site
Mainz A. Rhein
55131
Germany
Research Site
Marburg
35043
Germany
Research Site
München
81675
Germany
Research Site
Münster
48145
Germany
Research Site
Münster
48149
Germany
Research Site
Neubrandenburg
17036
Germany
Research Site
Regensburg
93053
Germany
Research Site
Tübingen
72076
Germany
Research Site
Ulm
89081
Germany
Research Site
Würzburg
97080
Germany
Research Site
Budapest
1133
Hungary
Research Site
Pécs
7621
Hungary
Research Site
Florence
50134
Italy
Research Site
Genova
16132
Italy
Research Site
Milan
20122
Italy
Research Site
Milan
20132
Italy
Research Site
Milan
20157
Italy
Research Site
Naples
80131
Italy
Research Site
Roma
00133
Italy
Research Site
Roma
00168
Italy
Research Site
Fukushima
960-1295
Japan
Research Site
Kita-gun
761-0793
Japan
Research Site
Osaka
5458586
Japan
Research Site
Riga
LV-1009
Latvia
Research Site
Riga
LV1002
Latvia
Research Site
Bratislava
811 08
Slovakia
Research Site
Žilina
012 07
Slovakia
Research Site
Busan
49241
South Korea
Research Site
Daegu
42415
South Korea
Research Site
Incheon
22332
South Korea
Research Site
Jinju
52727
South Korea
Research Site
Seoul
03082
South Korea
Research Site
Seoul
06351
South Korea
Research Site
Barcelona
08022
Spain
Research Site
Barcelona
08035
Spain
Research Site
Barcelona
8025
Spain
Research Site
Bilbao
48006
Spain
Research Site
Córdoba
14012
Spain
Research Site
Majadahonda
28222
Spain
Research Site
Pamplona
31005
Spain
Research Site
Seville
41071
Spain
Research Site
Valencia
46014
Spain
Research Site
Valladolid
46012
Spain
Research Site
Zaragoza
50009
Spain
Research Site
Bristol
BS1 2LX
United Kingdom
Research Site
Liverpool
L69 3BX
United Kingdom
Research Site
London
EC1V 2PD
United Kingdom
Research Site
London
GU16 7UJ
United Kingdom
Research Site
London
NW1 5QH
United Kingdom
Research Site
London
SE5 9RS
United Kingdom
Research Site
Newcastle upon Tyne
NE7 7DN
United Kingdom
Research Site
Southampton
S016 6YD
United Kingdom
FG002
Danicopan 400 mg QD
Participants received danicopan 400 mg once daily (QD) during the 104-week Masked Treatment Period.
FG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
FG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
FG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
FG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
FG00093 subjects
FG00191 subjects
FG00291 subjects
FG00390 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
Received at Least 1 Dose of Study Drug
FG00093 subjects
FG00191 subjects
FG00291 subjects
FG00390 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
COMPLETED
FG00073 subjects
FG00167 subjects
FG00273 subjects
FG00370 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
NOT COMPLETED
FG00020 subjects
FG00124 subjects
FG00218 subjects
FG00320 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
Type
Comment
Reasons
Adverse Event
FG0007 subjects
FG0019 subjects
FG0028 subjects
FG0034 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
Investigator's Discretion
FG0001 subjects
FG0011 subjects
FG0020 subjects
FG0032 subjects
FG004
Withdrawal by Subject
FG0007 subjects
FG00113 subjects
FG0026 subjects
FG0039 subjects
FG004
Lost to Follow-up
FG0001 subjects
FG0010 subjects
FG0021 subjects
FG0030 subjects
FG004
Death
FG0000 subjects
FG0011 subjects
FG0020 subjects
FG0033 subjects
FG004
Liver abnormalities meeting Hy's Law criteria
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
Other Than Specified
FG0003 subjects
FG0010 subjects
FG0023 subjects
FG0032 subjects
FG004
Secondary Evaluation Period (52 Weeks)
Type
Comment
Milestone Data
STARTED
FG00073 subjects
FG00167 subjects
FG00273 subjects
FG0030 subjects
FG00424 subjects
FG00523 subjects
FG00623 subjects
Received at Least 1 Dose of Study Drug
FG00073 subjects
FG00167 subjects
FG00273 subjects
FG0030 subjects
COMPLETED
FG00015 subjects
FG00114 subjects
FG00211 subjects
FG0030 subjects
FG004
NOT COMPLETED
FG00058 subjects
FG00153 subjects
FG00262 subjects
FG0030 subjects
FG004
Type
Comment
Reasons
Adverse Event
FG0002 subjects
FG0013 subjects
FG0022 subjects
FG003
Full Analysis Set (FAS) included all randomized participants who received at least 1 dose (full or partial) of study drug grouped by randomized treatment group. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
BG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
BG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
BG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
BG004
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00093
BG00191
BG00291
BG00390
BG004365
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG00077.0± 7.20
BG00176.0± 7.24
BG00277.8± 7.52
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG00068
BG00158
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0004
BG0011
BG002
Race/Ethnicity, Customized
Count of Participants
Participants
Title
Denominators
Categories
Race
Title
Measurements
Asian
BG0002
BG0011
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) who had any post-baseline FAF GA lesion area data at the Week 52 analysis. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
Posted
Least Squares Mean
Standard Error
mm/year
Baseline, Week 52
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
Units
Counts
Participants
OG00065
OG00162
OG00270
OG003
Title
Denominators
Categories
Title
Measurements
OG0000.465± 0.0289
OG0010.391± 0.0306
OG0020.443± 0.0301
OG003
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG003
Mixed Models Analysis
0.9205
Least square (LS) mean difference
0.058
Standard Error of the Mean
0.0413
2-Sided
90
-0.010
0.126
Superiority
OG001
OG003
Mixed Models Analysis
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline visit and post-baseline (Week 104) visit FAF GA lesion area data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
Posted
Mean
Standard Deviation
mm
Baseline, Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit FAF GA lesion area data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
Posted
Mean
Standard Deviation
mm^2
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
percentage of EZ total attenuation
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
percentage of EZ partial attenuation
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Secondary
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).
All participants in FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit ONL-RPE mean central 1 mm subfield thickness data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
micrometers (µm)
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Secondary
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.
All participants in FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit ONL-RPE mean central macular 2 mm thickness data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
µm
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit EZ-RPE macular volume data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
cubic millimeters (mm^3)
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Secondary
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.
All participants in FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit EZ-RPE mean central 1 mm subfield thickness data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
µm
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Secondary
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.
All participants in FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit EZ-RPE mean central macular 2 mm thickness data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
µm
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
percentage of EZ total attenuation
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
percentage of EZ partial attenuation
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
percentage of EZ total attenuation
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
percentage of EZ partial attenuation
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
Secondary
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).
All participants in FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit sub-RPE central 1 mm subfield mean thickness data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
µm
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Secondary
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.
All participants in FAS (randomized participants who received at least 1 dose [full/partial] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit sub-RPE central macular 2 mm subfield mean thickness data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
µm
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Secondary
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).
All participants in FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit panmacular sub-RPE volume data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
mm^3
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Secondary
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.
All participants in FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit macular total RPE attenuation data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
percentage of macular area
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Secondary
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.
All participants in FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit central 1 mm subfield total RPE attenuation data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories.
Posted
Mean
Standard Deviation
percentage of total RPE attenuation
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
Secondary
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.
All participants in FAS (all randomized participants who received at least 1 dose [full/partial] of study drug grouped by randomized treatment) with both baseline and postbaseline (Week 52 and Week 104) visit central macular 2 mm subfield total RPE attenuation data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed= participants evaluable for specified categories.
Posted
Mean
Standard Deviation
percentage of total RPE attenuation
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
Secondary
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).
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit BCVA score data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
Posted
Mean
Standard Deviation
letters
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit LLVA score data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
Posted
Mean
Standard Deviation
letters
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit LLD score data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
Posted
Mean
Standard Deviation
letters
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit reading speed data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
Posted
Mean
Standard Deviation
words per minute (wpm)
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
Secondary
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.
All participants in the FAS (all randomized participants who received at least 1 dose [full or partial] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit NEI VFQ-25 scores data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints.
Posted
Mean
Standard Deviation
units on a scale
Baseline, Week 52 and Week 104
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
Secondary
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.
Pharmacokinetic (PK) Analysis Set included all participants who received at least 1 dose (full or partial) of study drug and had at least 1 measurable concentration value. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint.
Posted
Mean
Standard Deviation
ng/mL
Day 1 and Week 52
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
Units
Secondary
Change From Baseline in Ex Vivo Serum Alternative Pathway (AP) Activity
Serum AP functional activity was measured by the Wieslab functional immunoassay method.
Pharmacodynamic (PD) Analysis Set included all participants who received at least 1 dose (full or partial) of study drug and had at least 1 measurable PD value. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint.
Posted
Mean
Standard Deviation
percentage of activity
Week 52 (pre-dose, 2 hours post-dose)
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Secondary
Change From Baseline in Plasma Bb Fragment of Complement Factor B (Bb) Concentration
PD Analysis Set included all participants who received at least 1 dose (full or partial) of study drug and had at least 1 measurable PD value. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Posted
Mean
Standard Deviation
µg/mL
Baseline, Week 52 (pre-dose)
ID
Title
Description
OG000
Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 104-week Masked Treatment Period.
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
Secondary
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.
Safety Set included all participants who received at least 1 dose (full or partial) of study drug grouped by treatment actually received. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
Posted
Count of Participants
Participants
Day 1 through Week 108
ID
Title
Description
OG000
Primary Evaluation Period: Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 52-week Primary Evaluation Period.
OG001
Time Frame
Day 1 through Week 108
Description
Safety Set included all participants who received at least 1 dose (full or partial) of study drug grouped by treatment actually received. 'Deaths' as the 'reason for study discontinuation' have been reported in the participant flow section and 'all deaths' have been reported in the 'All-cause mortality' section.
As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Primary Evaluation Period: Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 52-week Primary Evaluation Period.
0
93
14
93
44
93
EG001
Primary Evaluation Period: Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 52-week Primary Evaluation Period.
1
91
19
91
42
91
EG002
Primary Evaluation Period: Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 52-week Primary Evaluation Period.
0
91
16
91
33
91
EG003
Primary Evaluation Period: Placebo
Participants received matching placebo during the 52-week Primary Evaluation Period.
3
90
15
90
33
90
EG004
Secondary Evaluation Period: Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 52-week Secondary Evaluation Period.
1
73
8
73
22
73
EG005
Secondary Evaluation Period: Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 52-week Secondary Evaluation Period.
1
67
10
67
14
67
EG006
Secondary Evaluation Period: Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 52-week Secondary Evaluation Period.
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
0
23
4
23
8
23
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Macular hole
Eye disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG0031 affected90 at risk
EG0040 affected73 at risk
EG0050 affected67 at risk
EG0060 affected73 at risk
EG0070 affected24 at risk
EG0080 affected23 at risk
EG0090 affected23 at risk
Visual acuity reduced
Eye disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Iron deficiency anaemia
Blood and lymphatic system disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Acute myocardial infarction
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Atrial fibrillation
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Cardiac arrest
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Coronary artery disease
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Myocardial infarction
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Tachycardia
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Deafness unilateral
Ear and labyrinth disorders
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Obstructive pancreatitis
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Pancreatitis
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Rectal haemorrhage
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Induration
General disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Malaise
General disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Drug-induced liver injury
Hepatobiliary disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0021 affected91 at risk
EG003
Cholelithiasis
Hepatobiliary disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
COVID-19
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Bacterial sepsis
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
COVID-19 pneumonia
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Cellulitis
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Groin abscess
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Herpes zoster
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Influenza
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Pneumonia
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Respiratory syncytial virus infection
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Respiratory tract infection bacterial
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Sepsis
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Superinfection bacterial
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Skin laceration
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Femoral neck fracture
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Forearm fracture
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Ligament sprain
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Lower limb fracture
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Tibia fracture
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Hepatic enzyme increased
Investigations
MedDRA 27.1
Systematic Assessment
EG0002 affected93 at risk
EG0011 affected91 at risk
EG0022 affected91 at risk
EG003
Hypervolaemia
Metabolism and nutrition disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Intervertebral disc disorder
Musculoskeletal and connective tissue disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Intervertebral disc protrusion
Musculoskeletal and connective tissue disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Osteoarthritis
Musculoskeletal and connective tissue disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Invasive ductal breast carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Adenocarcinoma of colon
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Benign salivary gland neoplasm
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Intraductal proliferative breast lesion
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Lung neoplasm malignant
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Malignant melanoma stage IV
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Metastases to spine
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Myelodysplastic syndrome
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Prostatic adenoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Transitional cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Cerebrovascular accident
Nervous system disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Hypoxic-ischaemic encephalopathy
Nervous system disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Transient ischaemic attack
Nervous system disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Disorientation
Psychiatric disorders
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Nephrolithiasis
Renal and urinary disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Renal failure
Renal and urinary disorders
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Acute respiratory failure
Respiratory, thoracic and mediastinal disorders
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Hypoxia
Respiratory, thoracic and mediastinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0021 affected91 at risk
EG003
Organising pneumonia
Respiratory, thoracic and mediastinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Pulmonary embolism
Respiratory, thoracic and mediastinal disorders
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Hypertension
Vascular disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0021 affected91 at risk
EG003
Haematoma
Vascular disorders
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Hypertensive emergency
Vascular disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
Neovascular age-related macular degeneration
Eye disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Endophthalmitis
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Angina unstable
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Arrhythmia
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Cardiac failure
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Cardiac failure congestive
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Sinus node dysfunction
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Ventricular tachycardia
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Vertigo
Ear and labyrinth disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Gastrointestinal haemorrhage
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Large intestine polyp
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Pancreatitis acute
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Proctitis
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Asthenia
General disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Cholecystitis acute
Hepatobiliary disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Hepatic cirrhosis
Hepatobiliary disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Hydrocholecystis
Hepatobiliary disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Streptococcal bacteraemia
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Diabetic foot infection
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Gastrointestinal infection
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Ankle fracture
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Pelvic fracture
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Rib fracture
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Toxicity to various agents
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Traumatic arthrosis
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Upper limb fracture
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Electrocardiogram QT prolonged
Investigations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Haemoglobin decreased
Investigations
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Hypoglycaemia
Metabolism and nutrition disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Hypernatraemia
Metabolism and nutrition disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Hypokalaemia
Metabolism and nutrition disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Brain neoplasm malignant
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Colon cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Neuroendocrine carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Syncope
Nervous system disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Dizziness
Nervous system disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Device breakage
Product Issues
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Mental status changes
Psychiatric disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Renal colic
Renal and urinary disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Neovascular age-related macular degeneration
Eye disorders
MedDRA 27.1
Systematic Assessment
EG0005 affected93 at risk
EG0015 affected91 at risk
EG0025 affected91 at risk
EG0033 affected90 at risk
EG0043 affected73 at risk
EG0052 affected67 at risk
EG0061 affected73 at risk
EG0071 affected24 at risk
EG0081 affected23 at risk
EG0091 affected23 at risk
Nausea
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0005 affected93 at risk
EG0013 affected91 at risk
EG0024 affected91 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA 27.1
Systematic Assessment
EG0005 affected93 at risk
EG0011 affected91 at risk
EG0020 affected91 at risk
EG003
COVID-19
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0005 affected93 at risk
EG0017 affected91 at risk
EG0029 affected91 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0006 affected93 at risk
EG0016 affected91 at risk
EG0029 affected91 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA 27.1
Systematic Assessment
EG0009 affected93 at risk
EG0015 affected91 at risk
EG0023 affected91 at risk
EG003
Gamma-glutamyltransferase increased
Investigations
MedDRA 27.1
Systematic Assessment
EG0009 affected93 at risk
EG0017 affected91 at risk
EG0025 affected91 at risk
EG003
C-reactive protein increased
Investigations
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0013 affected91 at risk
EG0027 affected91 at risk
EG003
Hepatic enzyme increased
Investigations
MedDRA 27.1
Systematic Assessment
EG0005 affected93 at risk
EG0016 affected91 at risk
EG0024 affected91 at risk
EG003
Blood alkaline phosphatase increased
Investigations
MedDRA 27.1
Systematic Assessment
EG0004 affected93 at risk
EG0014 affected91 at risk
EG0025 affected91 at risk
EG003
Alanine aminotransferase increased
Investigations
MedDRA 27.1
Systematic Assessment
EG0005 affected93 at risk
EG0015 affected91 at risk
EG0021 affected91 at risk
EG003
Headache
Nervous system disorders
MedDRA 27.1
Systematic Assessment
EG0005 affected93 at risk
EG0014 affected91 at risk
EG0026 affected91 at risk
EG003
Hypertension
Vascular disorders
MedDRA 27.1
Systematic Assessment
EG0006 affected93 at risk
EG0014 affected91 at risk
EG0023 affected91 at risk
EG003
Cataract
Eye disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0013 affected91 at risk
EG0021 affected91 at risk
EG003
Atrial fibrillation
Cardiac disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0013 affected91 at risk
EG0020 affected91 at risk
EG003
Pyrexia
General disorders
MedDRA 27.1
Systematic Assessment
EG0000 affected93 at risk
EG0010 affected91 at risk
EG0020 affected91 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0002 affected93 at risk
EG0011 affected91 at risk
EG0022 affected91 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA 27.1
Systematic Assessment
EG0001 affected93 at risk
EG0013 affected91 at risk
EG0022 affected91 at risk
EG003
The study was terminated early because the primary efficacy endpoint was not met.
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG004
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00018
OG00113
OG00218
OG0036
OG0044
OG0054
Title
Denominators
Categories
Title
Measurements
OG0000.892± 0.4545
OG0010.705± 0.3831
OG0020.859± 0.3468
OG0030.668± 0.4866
OG0040.585± 0.1396
OG0050.787± 0.1858
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00065
OG00162
OG00270
OG00366
OG00424
OG00520
OG00622
Title
Denominators
Categories
Change at Week 52
ParticipantsOG00065
ParticipantsOG00162
ParticipantsOG00270
ParticipantsOG00366
ParticipantsOG00424
ParticipantsOG00520
ParticipantsOG00622
Title
Measurements
OG0002.347± 1.2972
OG0012.044± 1.2280
OG0022.274± 1.4482
OG003
Change at Week 104
ParticipantsOG00018
ParticipantsOG00113
ParticipantsOG00218
ParticipantsOG0030
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00164
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG0008.70± 4.681
OG0017.48± 4.226
OG0028.66± 4.633
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00116
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00164
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG0006.42± 4.917
OG0015.15± 3.806
OG0026.63± 4.599
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00116
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG000-4.60± 13.366
OG001-4.48± 13.398
OG002-4.86± 13.345
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG000-7.42± 7.354
OG001-6.56± 9.960
OG002-8.04± 8.589
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00164
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG000-0.09± 0.074
OG001-0.07± 0.047
OG002-0.09± 0.048
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00116
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG000-4.03± 3.888
OG001-3.48± 4.243
OG002-3.83± 4.044
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG000-3.45± 2.971
OG001-2.75± 2.633
OG002-3.20± 2.622
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG00016.73± 16.914
OG00115.93± 21.716
OG00218.68± 20.417
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG00010.31± 13.668
OG0018.24± 12.342
OG0029.51± 14.020
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG00016.04± 12.199
OG00113.61± 13.797
OG00216.10± 12.422
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG0008.24± 10.598
OG0016.09± 7.169
OG0027.54± 8.630
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG000-2.61± 5.838
OG001-2.89± 4.863
OG002-2.87± 5.842
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG000-2.75± 4.229
OG001-3.34± 3.314
OG002-3.02± 4.064
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00164
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG000-0.04± 0.052
OG001-0.04± 0.050
OG002-0.05± 0.059
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00116
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00164
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG0006.11± 3.874
OG0015.11± 2.706
OG0026.38± 3.718
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00116
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG00010.27± 13.204
OG0018.60± 11.277
OG0029.60± 13.794
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00072
OG00165
OG00272
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Study Eye: Change at Week 52
ParticipantsOG00069
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00368
ParticipantsOG00423
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG00011.07± 8.790
OG00110.86± 7.042
OG00211.21± 8.539
OG003
Study Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00117
ParticipantsOG00218
ParticipantsOG0030
Fellow Eye: Change at Week 52
ParticipantsOG00062
ParticipantsOG00146
ParticipantsOG00265
ParticipantsOG00360
Fellow Eye: Change at Week 104
ParticipantsOG00016
ParticipantsOG00111
ParticipantsOG00219
ParticipantsOG0030
Both Eyes Combined: Change at Week 52
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
ParticipantsOG00370
Both Eyes Combined: Change at Week 104
ParticipantsOG00019
ParticipantsOG00117
ParticipantsOG00219
ParticipantsOG0030
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00073
OG00168
OG00275
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Change at Week 52
ParticipantsOG00073
ParticipantsOG00168
ParticipantsOG00275
ParticipantsOG00370
ParticipantsOG00424
ParticipantsOG00523
ParticipantsOG00623
Title
Measurements
OG000-2.2± 8.56
OG001-2.0± 9.39
OG002-3.5± 11.35
OG003
Change at Week 104
ParticipantsOG00020
ParticipantsOG00117
ParticipantsOG00220
ParticipantsOG0030
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00074
OG00167
OG00272
OG00370
OG00424
OG00523
OG00622
Title
Denominators
Categories
Change at Week 52
ParticipantsOG00074
ParticipantsOG00167
ParticipantsOG00272
ParticipantsOG00370
ParticipantsOG00424
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG000-6.3± 11.61
OG001-4.6± 11.77
OG002-4.0± 11.23
OG003
Change at Week 104
ParticipantsOG00044
ParticipantsOG00141
ParticipantsOG00249
ParticipantsOG0030
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00074
OG00167
OG00271
OG00370
OG00424
OG00523
OG00622
Title
Denominators
Categories
Change at Week 52
ParticipantsOG00074
ParticipantsOG00167
ParticipantsOG00271
ParticipantsOG00370
ParticipantsOG00424
ParticipantsOG00523
ParticipantsOG00622
Title
Measurements
OG0004.3± 13.30
OG0012.6± 13.68
OG0020.3± 15.67
OG003
Change at Week 104
ParticipantsOG00044
ParticipantsOG00141
ParticipantsOG00248
ParticipantsOG0030
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00067
OG00162
OG00258
OG00356
OG00420
OG00515
OG00620
Title
Denominators
Categories
Change at Week 52
ParticipantsOG00067
ParticipantsOG00162
ParticipantsOG00258
ParticipantsOG00356
ParticipantsOG00420
ParticipantsOG00515
ParticipantsOG00620
Title
Measurements
OG000-137.36± 1016.889
OG001-19.80± 42.134
OG002-13.47± 77.927
OG003
Change at Week 104
ParticipantsOG00039
ParticipantsOG00137
ParticipantsOG00235
ParticipantsOG0030
OG001
Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 104-week Masked Treatment Period.
OG002
Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 104-week Masked Treatment Period.
OG003
Placebo
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
OG004
Placebo - Danicopan 100 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
OG005
Placebo - Danicopan 200 mg BID
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
OG006
Placebo - Danicopan 400 mg QD
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.
Units
Counts
Participants
OG00073
OG00167
OG00275
OG00370
OG00424
OG00523
OG00623
Title
Denominators
Categories
Change at Week 52
ParticipantsOG00073
ParticipantsOG00167
ParticipantsOG00275
ParticipantsOG00370
ParticipantsOG00424
ParticipantsOG00523
ParticipantsOG00623
Title
Measurements
OG000-0.425± 9.6019
OG001-5.816± 11.9162
OG002-4.456± 9.4963
OG003
Change at Week 104
ParticipantsOG00031
ParticipantsOG00129
ParticipantsOG00234
ParticipantsOG0030
Counts
Participants
OG00091
OG00187
OG00289
Title
Denominators
Categories
Day 1: Predose
ParticipantsOG00091
ParticipantsOG00187
ParticipantsOG00289
Title
Measurements
OG0000.4± 3.41
OG0010± 0
OG0020.2± 1.67
Day 1: 2 Hour Postdose
ParticipantsOG00090
ParticipantsOG00185
ParticipantsOG00288
Title
Measurements
OG000
Week 52: Predose
ParticipantsOG00070
ParticipantsOG00167
ParticipantsOG00272
Title
Measurements
OG000
Week 52: 2 Hour Postdose
ParticipantsOG00072
ParticipantsOG00165
ParticipantsOG00272
Title
Measurements
OG000
Participants received matching placebo and were re-randomized to one of the active treatment groups at Week 52.
Units
Counts
Participants
OG00067
OG00168
OG00270
OG00368
Title
Denominators
Categories
Change at Week 52: Predose
ParticipantsOG00064
ParticipantsOG00168
ParticipantsOG00268
ParticipantsOG00368
Title
Measurements
OG000-5.9± 17.49
OG001-21.3± 29.90
OG002-8.2± 24.48
OG003
Change at Week 52: 2 Hour Postdose
ParticipantsOG00067
ParticipantsOG00162
ParticipantsOG00270
ParticipantsOG00364
Units
Counts
Participants
OG00066
OG00165
OG00272
OG00370
Title
Denominators
Categories
Title
Measurements
OG000-0.3± 0.77
OG001-0.7± 0.94
OG002-0.2± 0.55
OG003-0.1± 0.55
Primary Evaluation Period: Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 52-week Primary Evaluation Period.
OG002
Primary Evaluation Period: Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 52-week Primary Evaluation Period.
OG003
Primary Evaluation Period: Placebo
Participants received matching placebo during the 52-week Primary Evaluation Period.
OG004
Secondary Evaluation Period: Danicopan 100 mg BID
Participants received danicopan 100 mg BID during the 52-week Secondary Evaluation Period.
OG005
Secondary Evaluation Period: Danicopan 200 mg BID
Participants received danicopan 200 mg BID during the 52-week Secondary Evaluation Period.
OG006
Secondary Evaluation Period: Danicopan 400 mg QD
Participants received danicopan 400 mg QD during the 52-week Secondary Evaluation Period.
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 100 mg BID for the remainder of the study.
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 200 mg BID for the remainder of the study.
Participants who received placebo during the primary evaluation period (up to Week 52), were re-randomized to receive danicopan 400 mg QD for the remainder of the study.