Study Stopped
Lack of efficacy.
A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study of Danicopan (ALXN2040) in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
2 other identifiers
interventional
365
13 countries
128
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Aug 2021
Typical duration for phase_2
128 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 19, 2021
CompletedStudy Start
First participant enrolled
August 23, 2021
CompletedFirst Posted
Study publicly available on registry
August 25, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 5, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
January 21, 2025
CompletedResults Posted
Study results publicly available
September 5, 2025
CompletedSeptember 5, 2025
August 1, 2025
2.9 years
August 19, 2021
July 2, 2025
August 15, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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 (28)
Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF
Baseline, Week 104
Change From Baseline to Week 52 and Week 104 in the Total GA Lesion Area in the Study Eye as Measured by FAF
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)
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
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
Baseline, Week 52 and Week 104
- +23 more secondary outcomes
Study Arms (4)
Danicopan: 100 mg
EXPERIMENTALParticipants 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.
Danicopan: 200 mg
EXPERIMENTALParticipants 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.
Danicopan: 400 mg
EXPERIMENTALParticipants 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.
Placebo
PLACEBO COMPARATORParticipants 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.
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (128)
Research Site
Phoenix, Arizona, 85020, United States
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Beverly Hills, California, 90211, United States
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Encino, California, 91436, United States
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Fresno, California, 93720, United States
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Fullerton, California, 92835, United States
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La Jolla, California, 92093, United States
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Pasadena, California, 91105, United States
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Sacramento, California, 95841, United States
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San Mateo, California, 94401, United States
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Walnut Creek, California, 94598, United States
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Colorado Springs, Colorado, 80909, United States
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Longmont, Colorado, 80503, United States
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Waterford, Connecticut, 06385, United States
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Clearwater, Florida, 33761, United States
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Coral Springs, Florida, 33067, United States
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Deerfield Beach, Florida, 33064, United States
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Naples, Florida, 34103, United States
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Palm Beach Gardens, Florida, 33418, United States
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St. Petersburg, Florida, 33711, United States
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Augusta, Georgia, 30909, United States
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New Albany, Indiana, 47150, United States
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West Des Moines, Iowa, 50266, United States
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Hagerstown, Maryland, 21740, United States
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Rosedale, Maryland, 21237, United States
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Towson, Maryland, 21204, United States
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Springfield, Massachusetts, 01107, United States
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Southaven, Mississippi, 38671, United States
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Reno, Nevada, 89502, United States
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Teaneck, New Jersey, 07666, United States
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Great Neck, New York, 11021, United States
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Durham, North Carolina, 27705, United States
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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
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Philadelphia, Pennsylvania, 19104-2640, United States
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Philadelphia, Pennsylvania, 19107, United States
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Greenville, South Carolina, 29605, United States
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Ladson, South Carolina, 29456, United States
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Rapid City, South Dakota, 57701, United States
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Austin, Texas, 78750, United States
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Bellaire, Texas, 77401, United States
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Conroe, Texas, 77384, United States
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Dallas, Texas, 75231, United States
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San Antonio, Texas, 78240, United States
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Southlake, Texas, 76092, United States
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Fairfax, Virginia, 22031, United States
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Lynchburg, Virginia, 24502, United States
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Adelaide, 5000, Australia
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Albury, 2640, Australia
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East Melbourne, 3002, Australia
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Hurstville, 2220, Australia
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Parramatta, 2150, Australia
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Strathfield, 2135, Australia
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Sydney, 2000, Australia
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Pardubice, 530 02, Czechia
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Prague, 150 00, Czechia
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Créteil, 94010, France
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Écully, 69130, France
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Lyon, 69004, France
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Marseille, 13008, France
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Nantes, 44000, France
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Nice, 06000, France
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Paris, 75010, France
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Paris, 75019, France
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Paris, 75571, France
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Poitiers, 86021, France
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Strasbourg, 67091, France
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Berlin, 12203, Germany
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Bonn, 53127, Germany
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Düsseldorf, 40225, Germany
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Freiburg im Breisgau, 79106, Germany
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Göttingen, 37075, Germany
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Hanover, 30625, Germany
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Heidelberg, 69120, Germany
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Mainz A. Rhein, 55131, Germany
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Marburg, 35043, Germany
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München, 81675, Germany
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Münster, 48145, Germany
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Münster, 48149, Germany
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Neubrandenburg, 17036, Germany
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Regensburg, 93053, Germany
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Tübingen, 72076, Germany
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Ulm, 89081, Germany
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Würzburg, 97080, Germany
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Budapest, 1133, Hungary
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Pécs, 7621, Hungary
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Florence, 50134, Italy
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Genova, 16132, Italy
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Milan, 20122, Italy
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Milan, 20132, Italy
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Milan, 20157, Italy
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Naples, 80131, Italy
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Roma, 00133, Italy
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Roma, 00168, Italy
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Fukushima, 960-1295, Japan
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Kita-gun, 761-0793, Japan
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Osaka, 5458586, Japan
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Riga, LV-1009, Latvia
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Riga, LV1002, Latvia
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Bratislava, 811 08, Slovakia
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Žilina, 012 07, Slovakia
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Busan, 49241, South Korea
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Daegu, 42415, South Korea
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Incheon, 22332, South Korea
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Jinju, 52727, South Korea
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Seoul, 03082, South Korea
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Seoul, 06351, South Korea
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Barcelona, 08022, Spain
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Barcelona, 08035, Spain
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Barcelona, 8025, Spain
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Bilbao, 48006, Spain
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Córdoba, 14012, Spain
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Majadahonda, 28222, Spain
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Pamplona, 31005, Spain
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Seville, 41071, Spain
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Valencia, 46014, Spain
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Valladolid, 46012, Spain
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Zaragoza, 50009, Spain
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Bristol, BS1 2LX, United Kingdom
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Liverpool, L69 3BX, United Kingdom
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London, EC1V 2PD, United Kingdom
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London, GU16 7UJ, United Kingdom
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London, NW1 5QH, United Kingdom
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London, SE5 9RS, United Kingdom
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Newcastle upon Tyne, NE7 7DN, United Kingdom
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Southampton, S016 6YD, United Kingdom
Related Publications (1)
Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.
PMID: 37314061DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Limitations and Caveats
The study was terminated early because the primary efficacy endpoint was not met.
Results Point of Contact
- Title
- Alexion Pharmaceuticals Inc.
- Organization
- Alexion Pharmaceuticals Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Masking Details
- Masking of treatment allocation will be observed until Week 104.
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 19, 2021
First Posted
August 25, 2021
Study Start
August 23, 2021
Primary Completion
July 5, 2024
Study Completion
January 21, 2025
Last Updated
September 5, 2025
Results First Posted
September 5, 2025
Record last verified: 2025-08