Ignite Creation Date:
2024-05-05 @ 11:52 AM
Last Modification Date:
2024-10-26 @ 9:15 AM
Study NCT ID:
NCT00158600
Status:
COMPLETED
Last Update Posted:
2015-04-28
First Post:
2005-09-08
Brief Title:
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme Alglucosidase Alfa in Patients With Late-Onset Pompe Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
Randomized Double-Blind Placebo-Controlled Study of the Safety Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease
Status:
COMPLETED
Status Verified Date:
2015-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
True
If Expanded Access, NCT#:
NCT00520143
Has Expanded Access, NCT# Status:
APPROVED_FOR_MARKETING
Acronym:
None
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 bodys 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:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2005-002759-42 | EUDRACT_NUMBER | None | None |