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

NCT ID: NCT05019521

Last Updated: 2025-09-05

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

365 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-08-23

Study Completion Date

2025-01-21

Brief Summary

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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

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Conditions

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Geographic Atrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Masking of treatment allocation will be observed until Week 104.

Study Groups

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Danicopan: 100 mg

Participants will receive danicopan 100 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Group Type EXPERIMENTAL

Danicopan

Intervention Type DRUG

Oral tablet.

Danicopan: 200 mg

Participants will receive danicopan 200 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Group Type EXPERIMENTAL

Danicopan

Intervention Type DRUG

Oral tablet.

Danicopan: 400 mg

Participants will receive danicopan 400 mg qd during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Group Type EXPERIMENTAL

Danicopan

Intervention Type DRUG

Oral tablet.

Placebo

Participants will receive matching placebo and will be re-randomized to one of the active treatment groups at Week 52, or to the optimal dose, if already identified.

Group Type PLACEBO_COMPARATOR

Danicopan

Intervention Type DRUG

Oral tablet.

Placebo

Intervention Type DRUG

Oral tablet.

Interventions

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Danicopan

Oral tablet.

Intervention Type DRUG

Placebo

Oral tablet.

Intervention Type DRUG

Other Intervention Names

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ALXN2040

Eligibility Criteria

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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

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.
Minimum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Phoenix, Arizona, United States

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Beverly Hills, California, United States

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Encino, California, United States

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Fresno, California, United States

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Fullerton, California, United States

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La Jolla, California, United States

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Pasadena, California, United States

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Sacramento, California, United States

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San Mateo, California, United States

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Walnut Creek, California, United States

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Colorado Springs, Colorado, United States

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Longmont, Colorado, United States

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Waterford, Connecticut, United States

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Clearwater, Florida, United States

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Coral Springs, Florida, United States

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Deerfield Beach, Florida, United States

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Naples, Florida, United States

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Palm Beach Gardens, Florida, United States

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St. Petersburg, Florida, United States

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Augusta, Georgia, United States

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New Albany, Indiana, United States

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West Des Moines, Iowa, United States

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Hagerstown, Maryland, United States

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Rosedale, Maryland, United States

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Towson, Maryland, United States

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Springfield, Massachusetts, United States

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Southaven, Mississippi, United States

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Reno, Nevada, United States

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Teaneck, New Jersey, United States

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Great Neck, New York, United States

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Durham, North Carolina, United States

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Cincinnati, Ohio, United States

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Portland, Oregon, United States

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Springfield, Oregon, United States

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Kingston, Pennsylvania, United States

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Monroeville, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Greenville, South Carolina, United States

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Ladson, South Carolina, United States

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Rapid City, South Dakota, United States

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Austin, Texas, United States

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Bellaire, Texas, United States

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Conroe, Texas, United States

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Dallas, Texas, United States

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San Antonio, Texas, United States

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Southlake, Texas, United States

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Fairfax, Virginia, United States

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Lynchburg, Virginia, United States

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Adelaide, , Australia

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Albury, , Australia

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East Melbourne, , Australia

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Hurstville, , Australia

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Parramatta, , Australia

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Strathfield, , Australia

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Sydney, , Australia

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Pardubice, , Czechia

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Prague, , Czechia

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Créteil, , France

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Écully, , France

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Lyon, , France

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Marseille, , France

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Nantes, , France

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Nice, , France

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Paris, , France

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Paris, , France

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Paris, , France

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Poitiers, , France

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Strasbourg, , France

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Berlin, , Germany

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Bonn, , Germany

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Düsseldorf, , Germany

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Freiburg im Breisgau, , Germany

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Göttingen, , Germany

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Hanover, , Germany

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Heidelberg, , Germany

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Mainz A. Rhein, , Germany

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Marburg, , Germany

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München, , Germany

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Münster, , Germany

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Münster, , Germany

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Neubrandenburg, , Germany

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Regensburg, , Germany

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Tübingen, , Germany

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Ulm, , Germany

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Würzburg, , Germany

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Budapest, , Hungary

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Pécs, , Hungary

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Florence, , Italy

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Genova, , Italy

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Milan, , Italy

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Milan, , Italy

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Milan, , Italy

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Naples, , Italy

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Roma, , Italy

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Roma, , Italy

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Fukushima, , Japan

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Kita-gun, , Japan

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Osaka, , Japan

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Riga, , Latvia

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Riga, , Latvia

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Bratislava, , Slovakia

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Žilina, , Slovakia

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Busan, , South Korea

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Daegu, , South Korea

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Incheon, , South Korea

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Jinju, , South Korea

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Seoul, , South Korea

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Seoul, , South Korea

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Barcelona, , Spain

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Barcelona, , Spain

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Barcelona, , Spain

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Bilbao, , Spain

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Córdoba, , Spain

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Majadahonda, , Spain

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Pamplona, , Spain

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Seville, , Spain

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Valencia, , Spain

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Valladolid, , Spain

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Zaragoza, , Spain

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Bristol, , United Kingdom

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Liverpool, , United Kingdom

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London, , United Kingdom

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London, , United Kingdom

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London, , United Kingdom

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London, , United Kingdom

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Newcastle upon Tyne, , United Kingdom

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Southampton, , United Kingdom

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Countries

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United States Australia Czechia France Germany Hungary Italy Japan Latvia Slovakia South Korea Spain United Kingdom

References

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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.

Reference Type DERIVED
PMID: 37314061 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2021-001198-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ALXN2040-GA-201

Identifier Type: -

Identifier Source: org_study_id

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