Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT07413250

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes PNH

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Clinical Research Site

Boston, Massachusetts, 02210, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Danicopan

Drug

Participants treated with danicopan as an add-on therapy.

Soliris/Ultomiris

Drug

Participants treated with Soliris/Ultomiris monotherapy.

Primary outcomes

  1. 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

  2. Event Rate of Meningococcal Infections

    Time frame: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years

  3. Event Rate of Serious Infections

    Time frame: Up to approximately 5 years

  4. Event Rate of Malignancies and Hematologic Abnormalities

    Time frame: Up to approximately 5 years

Secondary outcomes

  1. 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

  2. Number of Infant Health Abnormalities in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding

    Time frame: Up to 12 months

  3. Number of Participants with PNH symptoms at Diagnosis

    Time frame: Baseline (Day 1)

  4. Number of Participants with a History Bone Marrow Transplant

    Time frame: Baseline (Day 1)

  5. Number of Participants with a history of Major Adverse Vascular Events, including Thrombosis

    Time frame: Baseline (Day 1)

  6. Number of Participants with Ongoing Severe Hepatic Impairment as Defined by Child-Pugh Class C at Enrollment

    Time frame: Baseline (Day 1)

  7. Number of Participants Who Discontinue Danicopan Treatment

    Time frame: Up to approximately 5 years

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2030
Study completion
2030
First posted
Feb 17, 2026
Registry last updated
Feb 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.