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

February 10, 2026 updated by: Alexion Pharmaceuticals, Inc.

An Observational Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

50

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02210
        • Alexion Pharmaceuticals, Inc. (Sponsor)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The study population will consist of adult participants ≥ 18 years at danicopan treatment initiation (baseline) as add-on therapy to ravulizumab/eculizumab. Participants will have initiated treatment with danicopan on or after registry enrollment.

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Danicopan
Participants with PNH treated with Danicopan as an add-on therapy to eculizumab or ravulizumab.
Drug: Danicopan
Participants will receive Danicopan as an add-on therapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants with Serious Adverse Events (SAEs)
Time Frame: Up to approximately 5 years
Up to approximately 5 years
Number of Participants with Serious Infections Caused by Encapsulated Bacteria
Time Frame: Up to approximately 5 years
Up to approximately 5 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Number of Participants Who Discontinue Study Treatment
Time Frame: Up to approximately 5 years
Up to approximately 5 years
Frequency of Reasons for Discontinuation
Time Frame: Up to approximately 5 years
Up to approximately 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 18, 2025

Primary Completion (Estimated)

July 1, 2029

Study Completion (Estimated)

July 1, 2029

Study Registration Dates

First Submitted

February 10, 2026

First Submitted That Met QC Criteria

February 10, 2026

First Posted (Actual)

February 17, 2026

Study Record Updates

Last Update Posted (Actual)

February 17, 2026

Last Update Submitted That Met QC Criteria

February 10, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • D7332R00004
  • ALX-PNH-503 (Other Identifier: Alexion)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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