VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease
Status:
Terminated
Trial end date:
2020-03-25
Target enrollment:
Participant gender:
Summary
This Phase I/II open-label, randomized, dose-escalation study will assess the safety,
tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of VAL-1221 versus
Myozyme®/Lumizyme® in participants with late-onset glycogen storage disease-II (GSD-II)
(Pompe disease)