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

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

Recruiting
Phase of Trial: Phase I

Latest Information Update: 28 Sep 2017

At a glance

  • Drugs Hydroxypropyl betadex (Primary)
  • Indications Niemann-Pick disease type C
  • Focus Biomarker; Pharmacokinetics
  • Sponsors CTD Holdings
  • Most Recent Events

    • 28 Sep 2017 According to a CTD Holdings media release, first patient has been dosed and final data is expected in 2018.
    • 22 Sep 2017 According to a CTD Holdings media release, data from this trial will be presented at the Niemann-Pick UK 8th Interactive Workshop on Niemann-Pick Diseases and NPUK 24th Annual Family Conference.
    • 10 Jun 2017 Biomarkers information updated
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