Ignite Creation Date:
2024-05-05 @ 4:58 PM
Last Modification Date:
2024-10-26 @ 9:26 AM
Study NCT ID:
NCT00358150
Status:
COMPLETED
Last Update Posted:
2017-02-15
First Post:
2006-07-27
Brief Title:
A Study of the Efficacy and Safety of Eliglustat Tartrate Genz-112638 in Type 1 Gaucher Patients
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
A Phase 2 Open-Label Multi-Center Study Evaluating the Efficacy Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients
Status:
COMPLETED
Status Verified Date:
2016-12
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:
Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid beta-glucosidase also known as glucocerebrosidase This enzyme is needed to digest a substrate lipid called glucosylceramide and to a lesser degree glucosylsphingosine In participants with Gaucher disease the liver spleen bone marrow and brain show increases in lipid concentration specifically in cells derived from the monocytemacrophage system
Eliglustat tartrate Genz-112638 is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide The primary objective of this study is to evaluate the efficacy safety and pharmacokinetics PK of eliglustat tartrate administered as an oral dose of either 50 milligram mg twice daily BID or 100 mg BID to men and women with Gaucher disease Type 1 for 52 weeks
Eliglustat tartrate Genz-112638 is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide The primary objective of this study is to evaluate the efficacy safety and pharmacokinetics PK of eliglustat tartrate administered as an oral dose of either 50 milligram mg twice daily BID or 100 mg BID to men and women with Gaucher disease Type 1 for 52 weeks
Detailed Description:
This study consists of several phases screening -28 to -1 days dose adjustmenttreatment Day 1 treatment baseline to Day 30 initial steady-state treatment post-Day 30 through Week 52 post-baseline a treatment interruption period Week 52 through approximately Week 54 long-term steady-state treatment approximately Week 54 through study completion and safety follow-up 30 to 37 days after a participant withdraws from or completes the study The Primary Analysis Period is from baseline through Week 52 The Extension Period is from Week 52 through study completion that is participant withdrawal the study is terminated eliglustat tartrate becomes commercially available or where applicable specific regulatory requirements have been met
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2005-004732-42 | EUDRACT_NUMBER | None | None |
| DRI12816 | OTHER | Sanofi | None |