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

Running, not enrolling

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria, PNH

In brief

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.

Key facts

Study ID

NCT07413250

Run by

Alexion Pharmaceuticals, Inc.

People needed

50

Starts

2026-01-14

Expected to finish

2030-01-15

Last updated by the study team

2026-02-17

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Adult participants aged ≥ 18 years at treatment initiation.
• Initiated treatment with Ultomiris, Soliris, and/or danicopan on or after IPIG or Alexion International PNH Registry enrollment.

You may not qualify if…

• Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.

Where it is running

• Clinical Research Site — Boston, Massachusetts, United States

Full record on ClinicalTrials.gov

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