|Professor Volker Straub
|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