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

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

A Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of International PNH Interest Group (IPIG)-Registry Data

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.

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
        • Clinical Research Site

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 2 treatment cohorts:

  • Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment.
  • Participants initiating treatment with Ultomiris or Soliris on or after IPIG or Alexion International PNH Registry enrollment and without any danicopan treatment experience during follow-up.

Description

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.

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
Cohort 1
Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment.
Drug: Danicopan
Participants treated with danicopan as an add-on therapy.
Cohort 2
Participants initiating treatment with Ultomiris or Soliris on or after IPIG or Alexion International PNH Registry enrollment and without any danicopan treatment experience during follow-up.
Drug: Soliris/Ultomiris
Participants treated with Soliris/Ultomiris monotherapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
Event Rate of Meningococcal Infections
Time Frame: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years
Event Rate of Serious Infections
Time Frame: Up to approximately 5 years
Up to approximately 5 years
Event Rate of Malignancies and Hematologic Abnormalities
Time Frame: Up to approximately 5 years
Up to approximately 5 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Number of Participants with AEs and SAEs in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding
Time Frame: Up to approximately 5 years
Up to approximately 5 years
Number of Infant Health Abnormalities in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding
Time Frame: Up to 12 months
Up to 12 months
Number of Participants with PNH symptoms at Diagnosis
Time Frame: Baseline (Day 1)
Baseline (Day 1)
Number of Participants with a History Bone Marrow Transplant
Time Frame: Baseline (Day 1)
Baseline (Day 1)
Number of Participants with a history of Major Adverse Vascular Events, including Thrombosis
Time Frame: Baseline (Day 1)
Baseline (Day 1)
Number of Participants with Ongoing Severe Hepatic Impairment as Defined by Child-Pugh Class C at Enrollment
Time Frame: Baseline (Day 1)
Baseline (Day 1)
Number of Participants Who Discontinue Danicopan Treatment
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)

January 14, 2026

Primary Completion (Estimated)

January 15, 2030

Study Completion (Estimated)

January 15, 2030

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

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • D7332R00003
  • ALX-PNH-502 (Registry Identifier: Alexion)

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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