Ignite Creation Date:
2024-05-05 @ 4:58 PM
Last Modification Date:
2024-10-26 @ 9:26 AM
Study NCT ID:
NCT00351156
Status:
COMPLETED
Last Update Posted:
2010-08-19
First Post:
2006-07-10
Brief Title:
Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
Sponsor:
Amicus Therapeutics
Organization:
Amicus Therapeutics
Study Overview
Official Title:
A Multicenter Study to Evaluate and Characterize the Ex Vivo Effect of Pharmacological Chaperone Therapy in Blood Cell Lines Derived From Patients With Gaucher Disease
Status:
COMPLETED
Status Verified Date:
2010-08
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:
The purpose of this study is to learn more about Gaucher disease The information we collect from medical histories and a blood sample from people with Gaucher disease may help us pinpoint certain things that are different between people who have Gaucher disease and people who do not have Gaucher disease This information may be useful in the future to help find new treatments for Gaucher disease
Detailed Description:
This study is designed to evaluate the ex vivo response to pharmacological chaperone therapy by testing blood samples from previously treated and untreated patients with Gaucher disease The study will include patients with non-neuropathic Gaucher disease type I and neuropathic Gaucher disease types II andor III
All subjects will participate in one study visit Clinical information will be collected retrospectively from medical records Information collected will include Gaucher disease diagnosis and history medical history family history assessments of clinical severity and genotype A blood sample will be collected and various cells will be isolated for laboratory testing and research
All subjects will participate in one study visit Clinical information will be collected retrospectively from medical records Information collected will include Gaucher disease diagnosis and history medical history family history assessments of clinical severity and genotype A blood sample will be collected and various cells will be isolated for laboratory testing and research
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