A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
NCT ID: NCT05389449
Last Updated: 2025-12-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
80 participants
INTERVENTIONAL
2022-10-28
2026-07-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Danicopan
Participants will receive their last dose of danicopan from the parent study the night prior to Day 1 of this LTE study and will continue daily treatment with danicopan together with their background C5i therapy.
Danicopan
Oral tablet
Interventions
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Danicopan
Oral tablet
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patient is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol.
* Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors.
Exclusion Criteria
* Patient has been permanently discontinued from danicopan in the parent study for any reason other than enrollment into this LTE study.
* Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Research Site
Los Angeles, California, United States
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Chicago, Illinois, United States
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Baltimore, Maryland, United States
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Kansas City, Missouri, United States
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Long Island City, New York, United States
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Cleveland, Ohio, United States
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Dallas, Texas, United States
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Porto Alegre, , Brazil
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Rio de Janeiro, , Brazil
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Toronto, Ontario, Canada
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Brno, , Czechia
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Lille, , France
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Paris, , France
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Pessac, , France
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Pierre-Bénite, , France
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Athens, , Greece
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Thessaloniki, , Greece
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Haifa, , Israel
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Jerusalem, , Israel
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Avellino, , Italy
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Bassano del Grappa, , Italy
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Florence, , Italy
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Milan, , Italy
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Reggio Calabria, , Italy
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Roma, , Italy
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Bunkyō City, , Japan
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Kyoto, , Japan
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Osaka, , Japan
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Shibuya-ku, , Japan
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Tsukuba, , Japan
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Kota Kinabalu, , Malaysia
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Kuching, , Malaysia
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Miri, , Malaysia
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Gdansk, , Poland
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Seoul, , South Korea
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Seoul, , South Korea
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Suwon, , South Korea
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Badalona, , Spain
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Barcelona, , Spain
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Majadahonda, , Spain
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Seville, , Spain
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Bangkok, , Thailand
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Airdrie, , United Kingdom
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Leeds, , United Kingdom
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London, , United Kingdom
Countries
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Other Identifiers
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ALXN2040-PNH-303
Identifier Type: -
Identifier Source: org_study_id