NCT05389449

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

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 80 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

The total duration of the study will be up to 3 years. Eligible participants must complete all study assessments on the parent protocol before starting this study. All participants entering this study will receive danicopan as an add-on to a background C5i therapy. The only allowed C5i therapies are eculizumab and ravulizumab.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
DRUG	Danicopan	Oral tablet

Timeline

• Start date: 2022-10-28
• Primary completion: 2026-07-31
• Completion: 2026-07-31
• First posted: 2022-05-25
• Last updated: 2026-04-16

Locations

45 sites across 15 countries: United States, Brazil, Canada, Czechia, France, Greece, Israel, Italy, Japan, Malaysia, Poland, South Korea, Spain, Thailand, United Kingdom

Regulatory

• FDA-regulated drug study