Status:

ACTIVE_NOT_RECRUITING

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

Lead Sponsor:

Alexion Pharmaceuticals, Inc.

Conditions:

Paroxysmal Nocturnal Hemoglobinuria

Eligibility:

All Genders

Phase:

PHASE3

Brief Summary

This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5...

Detailed Description

The total duration of the study will be up to 3 years. Eligible participants must complete all study assessments on the parent protocol before starting this study. All participants entering this study...

Eligibility Criteria

Inclusion

  • Key
  • All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment.
  • Patient is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol.
  • Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors.
  • Key

Exclusion

  • Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant.
  • Patient has been permanently discontinued from danicopan in the parent study for any reason other than enrollment into this LTE study.
  • Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.

Key Trial Info

Start Date :

October 28 2022

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

July 31 2026

Estimated Enrollment :

80 Patients enrolled

Trial Details

Trial ID

NCT05389449

Start Date

October 28 2022

End Date

July 31 2026

Last Update

December 31 2025

Active Locations (45)

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Page 1 of 12 (45 locations)

1

Research Site

Los Angeles, California, United States, 90089

2

Research Site

Chicago, Illinois, United States, 60612

3

Research Site

Baltimore, Maryland, United States, 21231

4

Research Site

Kansas City, Missouri, United States, 64111