Ignite Creation Date:
2024-05-05 @ 11:29 AM
Last Modification Date:
2024-10-26 @ 9:08 AM
Study NCT ID:
NCT00051935
Status:
COMPLETED
Last Update Posted:
2014-02-05
First Post:
2003-01-17
Brief Title:
A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
Open-Label Pilot Study of the Safety Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase rhGAA as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II 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:
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 pharmacokinetics pharmacodynamics and efficacy of recombinant human acid alpha-glucosidase rhGAA as a potential enzyme replacement therapy for a pair of siblings with GSD-II To be eligible for this study a patient must have a confirmed diagnosis of GSD-II and have a sister or brother who also has a confirmed diagnosis of GSD-II
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