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A Phase I Study to Evaluate the Single and Multiple-dose Pharmacokinetics of Intravenous Trappsol Cyclo (HP-Beta-CD) in Patients With Niemann-Pick Disease Type C (NPC-1) and the Effects of Dosing Upon Biomarkers of NPC Disease

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Trial Profile

A Phase I Study to Evaluate the Single and Multiple-dose Pharmacokinetics of Intravenous Trappsol Cyclo (HP-Beta-CD) in Patients With Niemann-Pick Disease Type C (NPC-1) and the Effects of Dosing Upon Biomarkers of NPC Disease

Status: Completed
Phase of Trial: Phase I

Latest Information Update: 20 Aug 2024

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At a glance

  • Drugs Hydroxypropyl betadex (Primary)
  • Indications Niemann-Pick disease type C
  • Focus Pharmacokinetics; Proof of concept
  • Sponsors Cyclo Therapeutics

    • Most Recent Events

    • 18 Oct 2022 According to a Cyclo Therapeutics media release, data from the trial were published in the peer reviewed, official journal of the Society for Inherited Metabolic Disorders, Molecular Genetics and Metabolism. The manuscript is authored by Professor Caroline Hastings, MD.
    • 18 Oct 2022 Results presented 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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