Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide
Clinical Trials in the USA / NCT07413679
Active, not recruiting Observational

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

NCT07413679 · tracked via the Priya Life Science USA tracker
Sponsor
Alexion Pharmaceuticals, Inc
Phase
Observational
Started
2025-08-18
Last updated
2026-02-17

Condition(s) studied

Paroxysmal Nocturnal HemoglobinuriaPNH

Investigational drug(s) / intervention(s)

Danicopan →

Danicopan: Participants will receive Danicopan as an add-on therapy.

Study summary

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Number of Participants with Serious Adverse Events (SAEs) — Up to approximately 5 years
  • Number of Participants with Serious Infections Caused by Encapsulated Bacteria — Up to approximately 5 years

Trial sites (1)

FacilityCityRegionStatus
Alexion Pharmaceuticals, Inc. (Sponsor) Boston Massachusetts

More Alexion Pharmaceuticals, Inc. trials in the USA

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES) Active, not recruiting
A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab Recruiting
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy Recruiting
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa Active, not recruiting
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis. Recruiting

Other trials for the same condition

Home Reported Outcomes in PNH Recruiting
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data 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
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
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 NCT07413679 on ClinicalTrials.gov ↗ ← All trials in the USA