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A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria

Trial Profile

A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 03 Nov 2023

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At a glance

  • Drugs Danicopan (Primary)
  • Indications Paroxysmal nocturnal haemoglobinuria
  • Focus Proof of concept; Therapeutic Use
  • Sponsors Alexion AstraZeneca Rare Disease; Alexion Pharmaceuticals
  • Most Recent Events

    • 06 Nov 2019 Results released at the 61st Annual Meeting and Exposition of the American Society of Hematology
    • 06 Nov 2019 According to an Achillion Pharmaceuticals media release, data from this study will be presented at the 61stAmerican Society of Hematology (ASH) Annual Meeting.
    • 10 Jun 2019 Results presented in an Achillion Pharmaceuticals media release.
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