Ignite Creation Date:
2024-05-05 @ 5:01 PM
Last Modification Date:
2024-10-26 @ 9:27 AM
Study NCT ID:
NCT00376168
Status:
COMPLETED
Last Update Posted:
2018-10-04
First Post:
2006-09-12
Brief Title:
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Sponsor:
Pfizer
Organization:
Pfizer
Study Overview
Official Title:
A Phase III Multicenter Randomized Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase prGCD in Patients With Gaucher Disease
Status:
COMPLETED
Status Verified Date:
2018-09
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 the most prevalent lysosomal storage disorder is caused by mutations in the human glucocerebrosidase gene GCD leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside GlcCer in the cells of the monocyte-macrophage system
This is the second trial to utilize a recombinant active form of lysosomal enzyme glucocerebrosidase human prGCD which is expressed and purified in a bioreactor system from transformed carrot plant root cell line
This is the second trial to utilize a recombinant active form of lysosomal enzyme glucocerebrosidase human prGCD which is expressed and purified in a bioreactor system from transformed carrot plant root cell line
Detailed Description:
This will be a multi-center randomized double-blind parallel group dose-ranging trial to assess the safety and efficacy of prGCD in 30 untreated patients with Gaucher disease Patients will receive IV infusion of prGCD every two weeks at the selected medical center The duration of the study will be nine months At the end of the 9-month treatment period 20 visits 38 weeks eligible patients will be offered enrollment in an open-label extension study
There will be two treatment groups 15 patients in each treatment group
Treatment Group I 30 unitskg every 2 weeks Treatment Group II 60 unitskg every 2 weeks
All patients will have pharmacokinetic data collected over approximately 3 hours with frequent blood samples following the first and final doses of prGCD
There will be two treatment groups 15 patients in each treatment group
Treatment Group I 30 unitskg every 2 weeks Treatment Group II 60 unitskg every 2 weeks
All patients will have pharmacokinetic data collected over approximately 3 hours with frequent blood samples following the first and final doses of prGCD
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