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An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age

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Trial Profile

An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 25 Dec 2025

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At a glance

  • Drugs Iptacopan (Primary)
  • Indications Paroxysmal nocturnal haemoglobinuria
  • Focus Adverse reactions; Pharmacokinetics; Registrational
  • Sponsors Novartis Pharmaceuticals

Most Recent Events

  • 11 Dec 2025 New trial record

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