clinical trial · NCT06449001
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
Alexion Pharmaceuticals, Inc.·phase3·recruiting·n = 6
Paroxysmal Nocturnal HemoglobinuriaPNHExtravascular HemolysisDanicopan
brief 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).
started
Aug 11, 2025
primary completion
May 28, 2027
completion
Mar 10, 2028
last updated
Dec 23, 2025
official title
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol