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: 05 Oct 2017

At a glance

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

    • 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.
    • 18 Sep 2017 According to a CTD Holdings media release, the Israeli Ministry of Health (MOH) has approved Clinical Trial Application to conduct this trial at two sites in Israel: the Soroka University Medical Center in Beer Sheva and Emek Medical Center in Afula.company expects patients to be enrolled at both Israeli sites by the end of October 2017.
    • 19 Jul 2017 According to a CTD Holdings media release, first patient has been dosed intravenously in this study.
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