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A Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal Hemoglobinuria

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Trial Profile

A Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal Hemoglobinuria

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 31 Oct 2023

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At a glance

  • Drugs Pelecopan (Primary)
  • Indications Paroxysmal nocturnal haemoglobinuria
  • Focus Adverse reactions; First in man; Proof of concept
  • Sponsors BioCryst Pharmaceuticals

    • Most Recent Events

    • 12 Sep 2023 Results presented at the 2023 American College of Clinical Pharmacology Annual Meeting
    • 17 Jun 2022 Results assessing (n=6) long-term safety, tolerability, and effectiveness of BCX9930 from dose-ranging study (NCT04330534) and extension study (NCT04702568), presented at the 27th Congress of the European Haematology Association.
    • 17 Jun 2022 Results assessing long-term safety, tolerability, and effectiveness of BCX9930, presented at the 27th Congress of the European Haematology Association.

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