NCT06449001

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis

A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).

Conditions

• Paroxysmal Nocturnal Hemoglobinuria
• PNH
• Extravascular Hemolysis

Interventions

Type	Name	Description
DRUG	Danicopan	Participants will receive danicopan on a weight-based dosing regimen.

Timeline

• Start date: 2025-08-11
• Primary completion: 2027-05-28
• Completion: 2028-03-10
• First posted: 2024-06-07
• Last updated: 2025-12-23

Locations

4 sites across 3 countries: Canada, France, United Kingdom

Regulatory

• FDA-regulated drug study