Trials / Completed
CompletedNCT00250939
A Study of rhGAA in Patients With Late-Onset Pompe Disease
Single-center, Open-label Study of Safety, Pharmacokinetics and Efficacy of rhGAA in Patients With Late-Onset Pompe Disease
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 5 (actual)
- Sponsor
- Genzyme, a Sanofi Company · Industry
- Sex
- All
- Age
- 5 Years – 18 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, pharmacokinetics (PK) and efficacy of Myozyme treatment.
Conditions
- Pompe Disease (Late-onset)
- Glycogen Storage Disease Type II (GSD-II)
- Acid Maltase Deficiency Disease
- Glycogenosis 2
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Myozyme | 20 mg/kg qow |
Timeline
- Start date
- 2005-02-01
- Primary completion
- 2006-07-01
- Completion
- 2006-11-01
- First posted
- 2005-11-09
- Last updated
- 2014-02-06
Locations
1 site across 1 country: Netherlands
Source: ClinicalTrials.gov record NCT00250939. Inclusion in this directory is not an endorsement.