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

NCT07413250 · tracked via the Priya Life Science USA tracker

Sponsor

Alexion Pharmaceuticals, Inc.

Phase

Observational

Started

2026-01-14

Last updated

2026-02-17

Condition(s) studied

Paroxysmal Nocturnal HemoglobinuriaPNH

Investigational drug(s) / intervention(s)

DanicopanSoliris/Ultomiris

Danicopan: Participants treated with danicopan as an add-on therapy.

Soliris/Ultomiris: Participants treated with Soliris/Ultomiris monotherapy.

Study summary

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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

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.

Primary outcome measure(s)

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

Trial sites (1)

Facility	City	Region	Status
Clinical Research Site	Boston	Massachusetts

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Other trials for the same condition

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH Active, not recruiting

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria Active, not recruiting

A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors Active, not recruiting

Long-term Safety of Danicopan: IPIG Registry-based Cohort Study Active, not recruiting

A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07413250 on ClinicalTrials.gov ↗ ← All trials in the USA