CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 40 enrolled
Drug / intervention
GA-GCB (velaglucerase alfa)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/NCT00478647
NCT00478647Phase 3Completed
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A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase

Shire·interventional·Posted May 25, 2007·Updated Jun 10, 2021

In Brief

A Phase 3 clinical trial evaluating GA-GCB (velaglucerase alfa) for Gaucher Disease. Completed, enrolled 40 participants across 15 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 the 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 safety and efficacy of every other week dosing of GA-GCB (velaglucerase alfa) in participants with type 1 Gaucher disease who were previously treated with imiglucerase.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsGaucher Disease
CountriesIsrael, Poland, Spain, United Kingdom, United States
Collaborators--

Timeline

Phase 3CompletedFinished
200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedMay 25, 2007
Enrollment StartJul 25, 2007
Primary CompletionJun 26, 2009
TodayJul 2, 2026
Enrollment to primary: 1.9 yearsPosted 19.1 years ago

Interventions

GA-GCB (velaglucerase alfa)biological

15-60 U/kg, every other week via intravenous infusion