Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
Status:
Completed
Trial end date:
2015-02-01
Target enrollment:
Participant gender:
Summary
Primary Objective:
To evaluate the safety and tolerability of neoGAA in treatment naïve and alglucosidase alfa
treated late-onset Pompe disease patients.
Secondary Objective:
To evaluate the pharmacokinetics, pharmacodynamics of neoGAA in treatment naïve and
alglucosidase alfa treated late-onset Pompe disease patients.
To evaluate the effect of neoGAA on exploratory efficacy endpoints in treatment naïve and
alglucosidase alfa treated late-onset Pompe disease patients.