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Completed

NCT Number: NCT00705939

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

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.

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Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

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

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Taliglucerase alfa

Drug

Intravenous infusion every 2 weeks

Other names: Plant Cell Expressed Recombinant Human Glucocerebrosidase, prGCD

Primary outcomes

  1. Spleen Volume

    Time frame: Spleen Volume at Baseline and Months 12, 24, and 36

    Spleen volume measured by MRI

Secondary outcomes

  1. Liver Volume

    Time frame: Liver volume at Baseline and Months 12, 24 and 36

    Liver volume measured by MRI

  2. Hemoglobin

    Time frame: Hemoglobin at Baseline and Months 12, 24 and 36

  3. Platelet Count

    Time frame: Platelet count at Baseline and Months 12, 24 and 36

Other outcomes

  1. 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)

  2. 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).

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study 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

Important dates

Study start
2008
Primary completion
2012
Study completion
2013
First posted
Jun 27, 2008
Registry last updated
Oct 4, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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