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A Phase I/II Study to Evaluate the Safety and PK of iv Trappsol Cyclo (HP-β-CD) in Patients With Niemann-Pick Disease Type C NPC-1 and the Pharmacodynamic Effects of Treatment Upon Markers of Cholesterol Metabolism and Clinical Outcomes

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Trial Profile

A Phase I/II Study to Evaluate the Safety and PK of iv Trappsol Cyclo (HP-β-CD) in Patients With Niemann-Pick Disease Type C NPC-1 and the Pharmacodynamic Effects of Treatment Upon Markers of Cholesterol Metabolism and Clinical Outcomes

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 04 Jul 2023

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At a glance

  • Drugs Hydroxypropyl betadex (Primary)
  • Indications Niemann-Pick disease type C
  • Focus Biomarker; Pharmacokinetics; Registrational; Therapeutic Use
  • Sponsors Cyclo Therapeutics

    • Most Recent Events

    • 29 Jun 2023 According to a Cyclo Therapeutics media release, data from this study were published in official journal of Molecular Genetics and Metabolism.
    • 29 Jun 2023 Results published in a Cyclo Therapeutics Media Release.
    • 16 Aug 2022 According to a Cyclo Therapeutics media release, data from this study were presented at the 2022 NPC Patient and Family Conference hosted by the Australian NPC Disease Foundation.
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