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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

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Trial Profile

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 of Trial: Phase III

Latest Information Update: 03 Sep 2025

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At a glance

  • Drugs Danicopan (Primary) ; Eculizumab (Primary) ; Ravulizumab (Primary)
  • Indications Paroxysmal nocturnal haemoglobinuria
  • Focus Registrational; Therapeutic Use
  • Sponsors Alexion AstraZeneca Rare Disease

Most Recent Events

  • 28 Aug 2025 Planned End Date changed from 1 May 2028 to 10 Mar 2028.
  • 13 Aug 2025 Planned End Date changed from 10 Mar 2028 to 1 May 2028.
  • 13 Aug 2025 Planned End Date changed from 25 Feb 2028 to 1 May 2028.

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