Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data

This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. The primary objectives seek to characterize the long-term safety profile of danicopan as add-on therapy to ravulizumab/eculizumab in participants with PNH.

NCT Number	NCT07413250
Lead Sponsor	Alexion Pharmaceuticals, Inc.
Conditions	Paroxysmal Nocturnal Hemoglobinuria, PNH
Enrollment	50 participants
Start Date	2026-01-14
Primary Completion	2030-01-15 (estimated)
Study Completion	2030-01-15 (estimated)
Updated on ClinicalTrials.gov	2026-02-17