Ignite Creation Date:
2024-05-05 @ 5:21 PM
Last Modification Date:
2024-10-26 @ 9:30 AM
Study NCT ID:
NCT00430625
Status:
COMPLETED
Last Update Posted:
2021-06-29
First Post:
2007-02-01
Brief Title:
A Study of Gene-Activated Human Glucocerebrosidase GA-GCB Enzyme Replacement Therapy in Gaucher Disease
Sponsor:
Shire
Organization:
Takeda
Study Overview
Official Title:
A Multicenter Randomized Double-Blind Parallel Group Two-Dose Study of Gene-Activated Human Glucocerebrosidase GA-GCB Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease
Status:
COMPLETED
Status Verified Date:
2021-06
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 rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase GCB Due to this deficiency of functional GCB glucocerebroside accumulates within macrophages leading to cellular engorgement organomegaly and organ system dysfunction The purpose of this study is to evaluate the efficacy of every other week dosing of Gene-Activated Human Glucocerebrosidase GA-GCB velaglucerase alfa at doses of 45 and 60 Ukg in treatment-naïve patients with type 1 Gaucher disease
Detailed Description:
Type 1 Gaucher disease the most common form accounts for more than 90 of all cases and does not involve the Central Nervous System CNS Typical manifestations of type 1 Gaucher disease include hepatomegaly splenomegaly thrombocytopenia bleeding tendencies anemia hypermetabolism skeletal pathology growth retardation pulmonary disease and decreased quality of life Gene-Activated human glucocerebrosidase GA-GCB velaglucerase alfa is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme Velaglucerase alfa contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease This study was designed to determine the efficacy safety and pharmacokinetics of GA-GCB in men women and children with Type 1 Gaucher disease Each patients duration of treatment was 12 months
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