At a glance
ClinicalIndex Comparison Record- ✓Diagnosis of Pompe disease with deficient GAA activity ≤40% of normal mean AND 2 confirmed GAA gene mutations
- ✓Age ≥8 years at enrollment
- ✓Ability to ambulate 40 meters in 6 minutes on two consecutive days (use of assistive devices permitted)
- ✓FVC 30-80% predicted in upright position
- ✕Requires invasive ventilatory support
- ✕Requires noninvasive ventilatory support while awake and upright
- ✕Prior enzyme replacement therapy with GAA from any source
- ✕Use of investigational product within 30 days prior to enrollment or concurrent enrollment in another clinical study (unless approved by Genzyme)
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.
In Brief
A Phase 3 clinical trial evaluating alglucosidase alfa and Placebo for Pompe Disease (Late-onset) and 3 related conditions. Completed, enrolled 90 participants across 8 sites in 3 countries.
Detailed 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.
Study Details
Timeline
Interventions
IV infusion of 20mg/kg; qow for 78 weeks.
Placebo Comparator; qow for 78 weeks.