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An Open Label, Single Arm, Multiple Dose Study to Assess Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of LNP023 When Administered in Addition to Standard of Care (SoC) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis

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An Open Label, Single Arm, Multiple Dose Study to Assess Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of LNP023 When Administered in Addition to Standard of Care (SoC) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 05 Jan 2024

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At a glance

  • Drugs Iptacopan (Primary) ; Eculizumab
  • Indications Paroxysmal nocturnal haemoglobinuria
  • Focus Therapeutic Use
  • Sponsors Novartis Pharma A.G.; Novartis Pharmaceuticals

    • Most Recent Events

    • 13 Dec 2022 Results of integrated analysis of X2201 and X2204 studies evaluating dose exposure response relationships of biomarkers and efficacy measures to support dose selection for iptacopan presented at the 64th American Society of Hematology Annual Meeting and Exposition
    • 30 Mar 2022 Status changed from active, no longer recruiting to completed.
    • 18 Aug 2021 Status changed from recruiting to active, no longer recruiting.

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