Ignite Creation Date:
2024-05-05 @ 12:11 PM
Last Modification Date:
2024-10-26 @ 9:21 AM
Study NCT ID:
NCT00268944
Status:
COMPLETED
Last Update Posted:
2014-02-05
First Post:
2005-12-22
Brief Title:
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
Prospective Open-label Single-arm Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory Support
Status:
COMPLETED
Status Verified Date:
2014-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
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 overall objective is to evaluate the safety and efficacy of rhGAA in patients with advanced Late-onset 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