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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

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

Latest Information Update: 02 Oct 2019

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At a glance

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

    • 02 Oct 2019 According to an Amicus Therapeutics media release, results are also available in a presentation on the Amicus corporate website and will be highlighted during the Amicus Analyst Day on October 10, 2019.
    • 02 Oct 2019 According to an Amicus Therapeutics media release, results are being featured at the 24th International Annual Congress of the World Muscle Society in an oral platform presentation on Friday October 4, 2019 at 10:00am CEST (4:00am EDT).
    • 02 Oct 2019 Results published in Amicus Therapeutics Media Release
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