NCT05389449Active, not recruitingPhase 3
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study
Source: ClinicalTrials.gov · Last updated 18 Jun 2026
Brief summary (registry, in English)
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 80 (actual)
- First submitted
- 20 May 2022
- Start date
- 28 Oct 2022
- Primary completion date
- 24 Aug 2026
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- NCT number
- NCT05389449
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 80
- From 28 Oct 2022 to the primary completion date, 24 Aug 2026
- 44 sites in 15 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant.
Other
- 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.
Who the study is looking for
- All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment.
- 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.
Sex: All · Does not accept healthy volunteers
Sites (44)
Sites in registry order. Save your city in your profile to sort them by distance.
This page is information, not medical advice. Discuss any trial with your doctor.