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Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
Sponsor: Alexion Pharmaceuticals, Inc.
Summary
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.
Official title: A Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of International PNH Interest Group (IPIG)-Registry Data
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
OBSERVATIONAL
Enrollment
50
Start Date
2026-01-14
Completion Date
2030-01-15
Last Updated
2026-02-17
Healthy Volunteers
No
Conditions
Interventions
Danicopan
Participants treated with danicopan as an add-on therapy.
Soliris/Ultomiris
Participants treated with Soliris/Ultomiris monotherapy.
Locations (1)
Clinical Research Site
Boston, Massachusetts, United States