drugset / Trial / NCT00763932
Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies
NaSingle-groupOpen-labelTreatment
Summary
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line
Timeline
- Start
- 2003-04
- Primary completion
- 2006-06
- Completion
- 2006-07
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | alglucosidase alfa | Protein / enzyme biologic | 10 mg/kg | — |
| Subject | alglucosidase alfa | Protein / enzyme biologic | 20 mg/kg | — |
| Subject | alglucosidase alfa | Protein / enzyme biologic | 40 mg/kg | — |