Ignite Creation Date:
2024-05-05 @ 5:33 PM
Last Modification Date:
2024-10-26 @ 9:33 AM
Study NCT ID:
NCT00483379
Status:
COMPLETED
Last Update Posted:
2014-03-07
First Post:
2007-06-06
Brief Title:
High Dose or High Dose Frequency Study of Alglucosidase Alfa
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
An Exploratory Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen
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 objective of this exploratory study is to evaluate the safety and efficacy of alternative dosing regimens of alglucosidase alfa in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mgkg every other week after a minimum of 6 months treatment immediately prior to study entry
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