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OpenTrials
Active, Not Recruiting

NCT Number: NCT07413679

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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

Alexion Pharmaceuticals, Inc. (Sponsor)

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 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.

Treatment and study plan

Danicopan

Drug

Participants will receive Danicopan as an add-on therapy.

Primary outcomes

  1. Number of Participants with Serious Adverse Events (SAEs)

    Time frame: Up to approximately 5 years

  2. Number of Participants with Serious Infections Caused by Encapsulated Bacteria

    Time frame: Up to approximately 5 years

Secondary outcomes

  1. Number of Participants Who Discontinue Study Treatment

    Time frame: Up to approximately 5 years

  2. Frequency of Reasons for Discontinuation

    Time frame: Up to approximately 5 years

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2029
Study completion
2029
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.

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