Long-term Safety of Danicopan: IPIG Registry-based Cohort Study

Running, not enrolling

Conditions studied: Paroxysmal Nocturnal Hemoglobinuria, PNH

In brief

This is a noninterventional cohort study using primary and secondary data from the International PNH Interest Group (IPIG) PNH registry. It is designed to characterize the long-term safety and tolerability of danicopan as add-on therapy to eculizumab or ravulizumab in adult participants with PNH.

Key facts

Study ID

NCT07413679

Run by

Alexion Pharmaceuticals, Inc.

People needed

50

Starts

2025-08-18

Expected to finish

2029-07-01

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 danicopan treatment initiation (baseline) as add-on therapy to ravulizumab/eculizumab.
• Initiated treatment with danicopan on or after registry enrollment

You may not qualify if…

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

Where it is running

• Alexion Pharmaceuticals, Inc. (Sponsor) — Boston, Massachusetts, United States

Full record on ClinicalTrials.gov

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