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

JRCT ID: jRCT2031230248

Registered date:21/07/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Date of first enrollment: 21/07/2023
• Target sample size: 100
• Countries of recruitment: United States,Japan,Italy,Japan,United Kingdom,Japan
• Study type: Interventional
• Intervention(s): Drug: Danicopan

Outcome(s)

• Primary Outcome: Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
• Secondary Outcome: Change In Hemoglobin Values

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

- 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.

Exclude criteria

- 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.

Related Information

• Primary Sponsor: Sugita Yuko