Ignite Creation Date:
2024-05-05 @ 11:33 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00074958
Status:
COMPLETED
Last Update Posted:
2015-04-02
First Post:
2003-12-24
Brief Title:
A Study of Fabrazyme in Pediatric Patients With Fabry Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
A Multi-center Phase 2 Open-Label Study of Fabrazyme Recombinant Human a-Galactosidase A Replacement Therapy in Pediatric Patients With Fabry Disease
Status:
COMPLETED
Status Verified Date:
2015-03
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:
People with Fabry disease have an alteration in their genetic material DNA which causes a deficiency of the a-galactosidase A enzyme This enzyme helps to break down and remove certain types of fatty substances called glycolipids These glycolipids are normally present within the body in most cells In people with Fabry disease glycolipids build up in various tissues such as the liver kidney skin and blood vessels because a-galactosidase A is not present or is present in small quantities The build up of glycolipid levels also referred to as globotriaosylceramide or GL-3 in these tissues is thought to cause the clinical symptoms that are common to Fabry disease Symptoms commonly appear during childhood with pain in the hands and feet This study explored the safety efficacy and pharmacokinetics of Fabrazyme in pediatric patients aged between 7 and 15 years
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