Trials / Completed
CompletedNCT00158600
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease.
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 90 (actual)
- Sponsor
- Genzyme, a Sanofi Company · Industry
- Sex
- All
- Age
- 8 Years
- Healthy volunteers
- Not accepted
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.
Conditions
- Pompe Disease (Late-onset)
- Glycogen Storage Disease Type II (GSD-II)
- Acid Maltase Deficiency Disease
- Glycogenosis 2
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | alglucosidase alfa | IV infusion of 20mg/kg; qow for 78 weeks. |
| DRUG | Placebo | Placebo Comparator; qow for 78 weeks. |
Timeline
- Start date
- 2005-09-01
- Primary completion
- 2007-09-01
- Completion
- 2007-09-01
- First posted
- 2005-09-12
- Last updated
- 2015-04-28
- Results posted
- 2010-07-23
Locations
8 sites across 3 countries: United States, France, Netherlands
Source: ClinicalTrials.gov record NCT00158600. Inclusion in this directory is not an endorsement.