Ignite Creation Date:
2024-05-05 @ 11:58 AM
Last Modification Date:
2024-10-26 @ 9:17 AM
Study NCT ID:
NCT00196716
Status:
COMPLETED
Last Update Posted:
2015-04-03
First Post:
2005-09-12
Brief Title:
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
A Multicenter Open-label Study of Low Dose Maintenance Treatment of Fabrazyme Recombinant Human Alpha-Galactosidase A R-h Alpha-GAL Replacement Therapy in 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 alpha-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 alpha-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 trial is designed to evaluate the efficacy of a lower dose of Fabrazyme in patients who initially received 10 mgkg every 2 weeks of Fabrazyme by investigating if the achieved clearance of glycosphingolipid deposits in the vascular endothelium of the kidney can be maintained at a lower dose
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