Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

September 5, 2018 updated by: Pfizer

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

45

Phase

  • Phase 3

Expanded Access

No longer available outside the clinical trial. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Victoria
      • Parkville, Victoria, Australia
        • Bone Marrow Transplant Service, The Royal Melbourne Hospital
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X5
        • Mount Sinai Hospital
  • Chile
      • Santiago, Chile
        • Pontificia Universidad Catolica de Chile
  • Israel
      • Haifa, Israel, 31096
        • Rambam Medical Center
      • Jerusalem, Israel
        • Shaare Zedek Medical Center
  • South Africa
      • Morningside, South Africa, 2196
        • Morningside Medi-Clinic
  • Spain
      • Zaragoza, Spain, 50009
        • Hospital Universitario Miguel Servet
  • United Kingdom
      • Cambridge, United Kingdom
        • Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
      • London, United Kingdom, NW3 2QG
        • Royal Free Hospital
  • United States
    • Georgia
      • Decatur, Georgia, United States, 30033
        • Department of Human Genetics, Emory University School of Medicine
    • New York
      • New York, New York, United States, 10016
        • Neurogenetics, NYU at Rivergate

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Naive 30 Units/kg
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Names:
  • Plant Cell Expressed Recombinant Human Glucocerebrosidase
  • prGCD
Experimental: Naive 60 Units/kg
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Names:
  • Plant Cell Expressed Recombinant Human Glucocerebrosidase
  • prGCD
Experimental: Switchover
Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Names:
  • Plant Cell Expressed Recombinant Human Glucocerebrosidase
  • prGCD

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Spleen Volume
Time Frame: Spleen Volume at Baseline and Months 12, 24, and 36
Spleen volume measured by MRI
Spleen Volume at Baseline and Months 12, 24, and 36

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Liver Volume
Time Frame: Liver volume at Baseline and Months 12, 24 and 36
Liver volume measured by MRI
Liver volume at Baseline and Months 12, 24 and 36
Hemoglobin
Time Frame: Hemoglobin at Baseline and Months 12, 24 and 36
Hemoglobin at Baseline and Months 12, 24 and 36
Platelet Count
Time Frame: Platelet count at Baseline and Months 12, 24 and 36
Platelet count at Baseline and Months 12, 24 and 36

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Spleen Volume Multiples of Normal (MN)
Time Frame: Baseline and Months 12, 24, and 36
Spleen volume measured by MRI. Normal spleen volume is 2 mL/kg × body weight (kg)
Baseline and Months 12, 24, and 36
Liver Volume Multiples of Normal (MN)
Time Frame: Baseline and Months 12, 24 and 36
Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg).
Baseline and Months 12, 24 and 36

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2008

Primary Completion (Actual)

May 1, 2012

Study Completion (Actual)

August 1, 2013

Study Registration Dates

First Submitted

June 25, 2008

First Submitted That Met QC Criteria

June 25, 2008

First Posted (Estimate)

June 27, 2008

Study Record Updates

Last Update Posted (Actual)

October 4, 2018

Last Update Submitted That Met QC Criteria

September 5, 2018

Last Verified

September 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PB-06-003

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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