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An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria

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Trial Profile

An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 17 Jun 2025

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At a glance

  • Drugs Pegcetacoplan (Primary)
  • Indications Paroxysmal nocturnal haemoglobinuria
  • Focus Adverse reactions; Pharmacodynamics; Pharmacokinetics; Therapeutic Use
  • Sponsors Apellis Pharmaceuticals

Most Recent Events

  • 11 Jun 2025 Planned End Date changed from 1 Oct 2024 to 1 Dec 2028.
  • 11 Jun 2025 Planned primary completion date changed from 1 Apr 2024 to 1 Dec 2028.
  • 08 Jan 2024 The trial has been Discontinued in Czechia according to European Clinical Trials Database record.

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