Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Phase 3

Completed

• Conditions: Gaucher Disease
• Interventions: Drug: Taliglucerase alfa

• Registration Number: NCT00705939
• Lead Sponsor: Pfizer

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 an extension trial to Study NCT00376168 and NCT00712348.

Detailed Description

This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.

Study Timeline

• Study Start: 2008-06
• Study First Submitted: 2008-06-25
• Study First Submitted QC: 2008-06-25
• Study First Posted: 2008-06-27
• Primary Completion: 2012-05
• Study Completion: 2013-08
• Results First Submitted: 2014-04-30
• Results First Submitted QC: 2014-07-14
• Results First Posted: 2014-07-15
• Status Verified: 2018-09
• Last Update Submitted: 2018-09-05
• Last Update Posted: 2018-10-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 45

Inclusion Criteria

• Successful completion of Protocol PB-06-001
• The patient signs informed consent

Exclusion Criteria

• Currently taking another experimental drug for any condition
• Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
• Pregnant or nursing
• Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Naive 60 Units/kg	Taliglucerase alfa	Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Switchover	Taliglucerase alfa	Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)
Naive 30 Units/kg	Taliglucerase alfa	Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)

Primary Outcome Measures

Name	Time	Method
Spleen Volume	Spleen Volume at Baseline and Months 12, 24, and 36	Spleen volume measured by MRI

Secondary Outcome Measures

Name	Time	Method
Platelet Count	Platelet count at Baseline and Months 12, 24 and 36
Hemoglobin	Hemoglobin at Baseline and Months 12, 24 and 36
Liver Volume	Liver volume at Baseline and Months 12, 24 and 36	Liver volume measured by MRI

Trial Locations

Locations (11)

Pontificia Universidad Catolica de Chile; 🇨🇱 Santiago, Chile

Bone Marrow Transplant Service, The Royal Melbourne Hospital; 🇦🇺 Parkville, Victoria, Australia

Department of Human Genetics, Emory University School of Medicine; 🇺🇸 Decatur, Georgia, United States

Rambam Medical Center; 🇮🇱 Haifa, Israel

Hospital Universitario Miguel Servet; 🇪🇸 Zaragoza, Spain

Royal Free Hospital; 🇬🇧 London, United Kingdom

Neurogenetics, NYU at Rivergate; 🇺🇸 New York, New York, United States

Shaare Zedek Medical Center; 🇮🇱 Jerusalem, Israel

Mount Sinai Hospital; 🇨🇦 Toronto, Ontario, Canada

Morningside Medi-Clinic; 🇿🇦 Morningside, South Africa

Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust; 🇬🇧 Cambridge, United Kingdom