Queen Square Centre for Neuromuscular Diseases


A study of neoGAA in naïve and alglucosidasealfa treated late-onset Pompe disease patients.

Sponsor Genzyme
Funder Genzyme
CI Professor Volker Straub
sites Newcastle
Contact Gillian.Kenyon@newcastle.ac.uk
More information on the trial

This is a phase I, multi-centre, multinational, open-label study of the drug neoGAA.

NeoGAA will be given in increasing doses and bi-weekly intravenous infusion.

  • Group 1 - Late-onset Pompe disease patients naïve to treatment, 3 dose levels
  • Group 2 - Late-onset Pompe disease patients previously treated with alglucosidasealfa, 3 dose levels


  • the safety and tolerability of neoGAA
  • the pharmacokinetic parameters of neoGAA
  • the pharmacodynamic effects of neoGAA on skeletal muscle and other exploratory biomarkers
  • the effect of neoGAA on exploratory efficacy endpoints