Conditions: Pompe Disease (Late-onset) Glycogen Storage Disease Type II (GSD-II) Acid Maltase Deficiency Disease Glycogenosis 2
Keywords: Glycogen Storage Disease Type II GSD-II Pompe Disease
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 90
Healthy volunteers: no

Brief summary:
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective is to evaluate the safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa treatment in patients with late-onset Pompe disease as compared to placebo.

Detailed description:

Arms:
arm 1: Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks. arm 2: Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks.

Interventions: intervention 1: alglucosidase alfa intervention 2: Placebo
Intervention descriptions: intervention 1: IV infusion of 20mg/kg; qow for 78 weeks. intervention 2: Placebo Comparator; qow for 78 weeks.

Protocol document: