An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease

Trial Profile

An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 29 Jun 2017

At a glance

  • Drugs ATB 200 (Primary) ; Miglustat
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions; First in man; Pharmacokinetics
  • Sponsors Amicus Therapeutics
  • Most Recent Events

    • 15 May 2017 Results published in an Amicus Therapeutics media release.
    • 01 Mar 2017 Status changed from recruiting to active, no longer recruiting, according to an Amicus Therapeutics media release.
    • 01 Mar 2017 According to an Amicus Therapeutics media release, data from this trial in additional naive and non-ambulatory patients, as well as extension-phase data on all patient cohorts, are expected in the second and third quarter of 2017.
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