Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Gaucher Disease Clinical Trial

Official title:

A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00705939
• Other study ID #: PB-06-003
• Status: Completed
• Phase: Phase 3
• Start date: June 2008
• Est. completion date: August 2013

Study information

• Verified date: September 2018
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 45
• Est. completion date: August 2013
• Est. primary completion date: May 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Gaucher Disease

Intervention

Drug:
• Taliglucerase alfa
Intravenous infusion every 2 weeks

Locations

Country	Name	City	State
Australia	Bone Marrow Transplant Service, The Royal Melbourne Hospital	Parkville	Victoria
Canada	Mount Sinai Hospital	Toronto	Ontario
Chile	Pontificia Universidad Catolica de Chile	Santiago
Israel	Rambam Medical Center	Haifa
Israel	Shaare Zedek Medical Center	Jerusalem
South Africa	Morningside Medi-Clinic	Morningside
Spain	Hospital Universitario Miguel Servet	Zaragoza
United Kingdom	Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust	Cambridge
United Kingdom	Royal Free Hospital	London
United States	Department of Human Genetics, Emory University School of Medicine	Decatur	Georgia
United States	Neurogenetics, NYU at Rivergate	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Chile,  Israel,  South Africa,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Spleen Volume Multiples of Normal (MN)	Spleen volume measured by MRI. Normal spleen volume is 2 mL/kg × body weight (kg)	Baseline and Months 12, 24, and 36
Other	Liver Volume Multiples of Normal (MN)	Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg).	Baseline and Months 12, 24 and 36
Primary	Spleen Volume	Spleen volume measured by MRI	Spleen Volume at Baseline and Months 12, 24, and 36
Secondary	Liver Volume	Liver volume measured by MRI	Liver volume at Baseline and Months 12, 24 and 36
Secondary	Hemoglobin		Hemoglobin at Baseline and Months 12, 24 and 36
Secondary	Platelet Count		Platelet count at Baseline and Months 12, 24 and 36