An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naive and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients

Trial Profile

An Open-label, Multicenter, Multinational, Ascending Dose Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Repeated Biweekly Infusions of neoGAA in naive and Alglucosidase Alfa Treated Late-onset Pompe Disease Patients

Completed
Phase of Trial: Phase I

Latest Information Update: 21 Apr 2016

At a glance

  • Drugs GZ 402666 (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions
  • Acronyms NEO1
  • Sponsors Genzyme Corporation; Sanofi
  • Most Recent Events

    • 07 Jun 2017 Biomarkers information updated
    • 21 Apr 2016 Results (n=21) assessing safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and exploratory efficacy presented at the 68th Annual Meeting of the American Academy of Neurology
    • 21 Apr 2016 Exploratory efficacy results (n=21) presented at the 68th Annual Meeting of the American Academy of Neurology
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top