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NCT Number: NCT00376168

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD 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 the second trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD) which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.

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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

Mount Sinai Hospital, Toronto, Ontario, Canada

Loading trial locations.

About this study

This will be a multi-center, randomized, double-blind, parallel group, dose-ranging trial to assess the safety and efficacy of prGCD in 30 untreated patients with Gaucher disease. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the study will be nine months. At the end of the 9-month treatment period (20 visits, 38 weeks) eligible patients will be offered enrollment in an open-label extension study.

There will be two treatment groups, 15 patients in each treatment group.

Treatment Group I: 30 units/kg every 2 weeks. Treatment Group II: 60 units/kg every 2 weeks.

All patients will have pharmacokinetic data collected over approximately 3 hours with frequent blood samples following the first and final doses of prGCD.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Males and females, 18 years or older
  • Confirmed enzymatic diagnosis of Gaucher disease
  • Splenomegaly defined as greater than eight times the expected volume (measured volume divided by estimated volume (0.2% of body weight)] as determined by MRI volumetric analysis
  • Female patients of child-bearing potential who agree to use a medically acceptable method of contraception
  • Thrombocytopenia (defined as platelet counts below the lower limit of normal) and/or anemia (defined by hemoglobin level at least 1 g/dL below normal range according to sex and age).
  • Patients who have not received ERT in the past or patients whoc have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody test.
  • Patients who have not received substrate reduction therapy (SRT) in the past 12 months.
  • Ability to provide a written informed consent.

Exclusion criteria

  • Currently taking another experimental drug for any condition
  • Pregnant or nursing
  • Presence of HIV and/or, HBsAg and/or hepatitis C infections
  • Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease.
  • Previous anaphylactoid reaction to Cerezyme® or Ceredase®.
  • History of allergy to carrots.

Treatment and study plan

Plant cell expressed recombinant glucocerebrosidase (prGCD)

Drug

Intravenous infusion every two weeks for 9 months

Other names: Taliglucerase alfa

Primary outcomes

  1. Change From Baseline in Spleen Volume Measured by MRI.

    Time frame: Baseline and 9 months

    Calculated as percent change in spleen volume from Baseline to 9 months

Secondary outcomes

  1. Change From Baseline in Liver Volume

    Time frame: Baseline and 9 months

    Calculated as percent change in liver volume from Baseline to 9 months

  2. Change in Hemoglobin

    Time frame: Baseline and Month 9

    Absolute change in Hemoglobin concentration from Baseline to Month 9

  3. Change in Platelet Count

    Time frame: Baseline and Month 9

    Change in Platelet count from Baseline to Month 9

Other outcomes

  1. Change in Chitotriosidase

    Time frame: Baseline and Month 9

    Change in Chitotriosidase from Baseline to Month 9

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study title

A Phase III, Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

Important dates

Study start
2007
Primary completion
2009
Study completion
2009
First posted
Sep 14, 2006
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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