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: 04 Oct 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

    • 04 Oct 2017 Based on the data from this trial, the company is planning discussions with regulators in the US and EU, and expects to provide an update in the first half of 2018, according to an Amicus Therapeutics media release.
    • 04 Oct 2017 Results from this trial were featured at the 22nd International Congress of the World Muscle Society, according to an Amicus Therapeutics media release.
    • 04 Oct 2017 Results published in an Amicus Therapeutics Media Release
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