Ignite Creation Date:
2024-05-05 @ 11:33 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00074919
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2014-02-05
First Post:
2003-12-23
Brief Title:
Expanded Access Use of Myozyme Alglucosidase Alfa in Patients With Infantile-onset Pompe Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
Expanded Access Use of Myozyme Alglucosidase Alfa in Patients With Infantile-onset Pompe Disease
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:
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 protocol is to provide enzyme replacement therapy with rhGAA on an expanded access basis to severely affected patients with infantile-onset Pompe disease for whom there is no alternative treatment and who do not meet the clinical characteristics described in the inclusion criteria for participation in other Genzyme Corporation-sponsored study currently enrolling patients with infantile-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