Ignite Creation Date:
2025-12-24 @ 1:18 PM
Ignite Modification Date:
2026-01-03 @ 11:05 PM
Study NCT ID:
NCT00455195
Status:
COMPLETED
Last Update Posted:
2014-03-07
First Post:
2007-03-30
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Late-Onset Treatment Study Extension Protocol
Sponsor:
Genzyme, a Sanofi Company
Organization:
Study Overview
Official Title:
An Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704
Status:
COMPLETED
Status Verified Date:
2014-02
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 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 objective of this extension study is to assess the long-term safety and efficacy of alglucosidase alfa treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704 (NCT00158600).
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
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?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 2006-003644-31 | EUDRACT_NUMBER | None | View |