Ignite Creation Date:
2024-05-05 @ 11:44 AM
Last Modification Date:
2024-10-26 @ 9:12 AM
Study NCT ID:
NCT00113035
Status:
COMPLETED
Last Update Posted:
2015-05-05
First Post:
2005-06-03
Brief Title:
Screening Protocol to Evaluate Acid Alpha-Glucosidase GAA Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
Screening Protocol to Evaluate Acid Alpha Glucosidase GAA Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease for Potential Inclusion in Future Clinical Studies With Myozyme Alglucosidase Alfa
Status:
COMPLETED
Status Verified Date:
2015-05
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:
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 primary objective of this study is to identify potential candidates for future clinical studies in Pompe disease
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