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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

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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

Status: Completed
Phase of Trial: Phase I

Latest Information Update: 15 Dec 2023

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At a glance

  • Drugs Avalglucosidase alfa (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions; Biomarker
  • Acronyms NEO1
  • Sponsors Genzyme Corporation; Sanofi

Most Recent Events

  • 01 Dec 2023 Population PK (PopPK) analysis was performed using nonlinear mixed effect modeling approach on pooled data from three clinical trials (NCT01898364, NCT02032524 and NCT02782741) with LOPD patients, and a phase 2 study (NCT03019406) with infantile-onset Pompe disease patients to propose alternative dosing regimens for pediatric LOPD patients based on a bodyweight cut-off,, published in the Journal of Pharmacokinetics and Pharmacodynamics.
  • 26 May 2022 Results of an analysis of data up to 6.5 years experience with Avalglucosidase alfa during the NEO1 and NEO-EXT studies published in the Neurology
  • 26 May 2020 Results presented at the 6th Congress of the European Academy of Neurology

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