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
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Registration Number
- JPRN-jRCT2031230248
- Lead Sponsor
- Sugita Yuko
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 100
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.
- 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.
- 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
- Secondary Outcome Measures
Name Time Method Change In Hemoglobin Values