Late-Onset Treatment Study Extension Protocol
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II
About this trial
This is an interventional treatment trial for Pompe Disease (Late-Onset)
Eligibility Criteria
Inclusion Criteria:
- Patient must have completed protocol AGLU02704 (NCT00158600)
- Patient must provide signed, informed consent prior to performing any study-related procedures
- Patient (and patient's legal guardian if patient is under 18 years of age) must have the ability to comply with the clinical protocol
- A female patient of childbearing potential must have a negative pregnancy test at Baseline. (note: all female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.)
Exclusion Criteria:
- The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, would preclude treatment with alglucosidase alfa.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Alglucosidase Alfa/Alglucosidase Alfa
Placebo/Alglucosidase Alfa
Participants who received alglucosidase alfa during the double-blind study and, if they completed the double-blind study, continued that treatment during the extension study. Participants received an intravenous (IV) infusion of 20 mg/kg of alglucosidase alfa every other week (qow) until their participation in both the AGLU02704 (NCT00158600) and AGLU03206 studies combined equaled a minimum of 104 weeks.
Participants given placebo during the double-blind study, completed the double-blind study (study AGLU02704, NCT00158600), and qualified to continue into the extension study on alglucosidase alfa. Participants received an intravenous (IV) infusion of 20 mg/kg of alglucosidase alfa every other week (qow) for up to 52 weeks. Only the alglucosidase alfa treatment experience is included in this extension study.