CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 25 enrolled
Drug / intervention
VPRIV ®,biological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00430625
NCT00430625Phase 3Completed
CompareCT.gov

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

Shire·interventional·Posted Feb 2, 2007·Updated Jun 29, 2021

In Brief

A Phase 3 clinical trial evaluating VPRIV ®, for Gaucher Disease, Type 1. Completed, enrolled 25 participants across 5 sites in 5 countries.

Detailed 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 U/kg in treatment-naïve patients with type 1 Gaucher disease.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsGaucher Disease, Type 1
CountriesArgentina, Israel, Paraguay, Russia, Tunisia
Collaborators--

Timeline

Phase 3CompletedFinished
200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedFeb 2, 2007
Enrollment StartFeb 15, 2007
Primary CompletionApr 1, 2009
TodayJul 2, 2026
Enrollment to primary: 2.1 yearsPosted 19.4 years ago

Interventions

VPRIV ®,biological

Intravenous (IV) infusion, every other week via intravenous infusion for 12 months