Ignite Creation Date:
2024-05-05 @ 6:39 PM
Last Modification Date:
2024-10-26 @ 9:35 AM
Study NCT ID:
NCT00520143
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2014-02-06
First Post:
2007-08-21
Brief Title:
Alglucosidase Alfa Temporary Access Program
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
Alglucosidase Alfa Temporary Access Program
Status:
APPROVED_FOR_MARKETING
Status Verified Date:
2014-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ATAP
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 objective of this expanded access study is to provide patients with Pompe disease in the United States US access to alglucosidase alfa produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration
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