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

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

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 19 Jul 2017

At a glance

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

    • 19 Jul 2017 According to a CTD Holdings media release, first patient has been dosed intravenously in this study.
    • 21 Jun 2017 According to a CTD Holdings media release, first patient has been enrolled and final data from this trial is expected by the end of 2018.
    • 10 Jun 2017 Biomarkers information updated
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