Ignite Creation Date:
2024-05-05 @ 11:29 AM
Last Modification Date:
2024-10-26 @ 9:08 AM
Study NCT ID:
NCT00053573
Status:
COMPLETED
Last Update Posted:
2014-02-05
First Post:
2003-01-31
Brief Title:
rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II Pompe Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
An Open-Label Multicenter Multinational Study of the Safety Efficacy Pharmacokinetics and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II
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:
Glycogen Storage Disease Type II GSD-II also known as Pompe disease 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 GSD-II an excessive amount of glycogen accumulates and is stored in various tissues especially heart and skeletal muscle which prevents their normal function This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase rhGAA as a potential enzyme replacement therapy for GSD-II Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old but less than or equal to 36 months old will be studied
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