Clinical Trial: VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Patients With Late-Onset Pompe Disease

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional




Official Title: A Three-Month, Randomized, Parallel Active Control, Single and Repeat Dose, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 D

Brief Summary: Phase I/II study enrolling up to 16 patients in a three-month, Randomized, Parallel Active Control, Single and Repeat Dose, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VAL-1221 Delivered Intravenously (IV) in Ambulatory and Ventilator-free Patients with Late-Onset GSD-II (Pompe Disease) Study