Clinical Trial

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

Recruiting Phase 3
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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).
Protocol Amendment History 9 amendments
This ClinicalTrials.gov record has been amended 9 times since 2024-06-03; most recent amendment 2025-12-19.
Status change: Not Yet Recruiting → Recruiting 2025-03-05
Trial Details
NCT Number NCT06449001
Lead Sponsor Alexion Pharmaceuticals, Inc.
Collaborators: AstraZeneca
Conditions Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
Enrollment 6 participants
Start Date 2025-08-11
Primary Completion 2027-05-28 (estimated)
Study Completion 2028-03-10 (estimated)
Updated on ClinicalTrials.gov 2025-12-23