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

Study Identifier:
D7332R00003
ClinicalTrials.gov Identifier:
NCT07413250
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Recruitment Complete

Study Details

Medical Condition
  • Paroxysmal Nocturnal Hemoglobinuria
  • PNH
Study Drug
  • Drug: Danicopan
  • Drug: Soliris/Ultomiris
Date
Jan 2026 - Jan 2030
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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.
Exclusion Criteria
  • Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.
Sex
Female & Male
Age
18+ years

Study Details

Medical Condition
  • Paroxysmal Nocturnal Hemoglobinuria
  • PNH
Study Drug
  • Drug: Danicopan
  • Drug: Soliris/Ultomiris
Date
Jan 2026 - Jan 2030
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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.
Exclusion Criteria
  • Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.

Protocol 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.

Trial Locations

Location
Status
Location
Clinical Research Site
Boston, Massachusetts, United States, 02210
Status
N/A