Skip to main content
OpenTrials
Completed

NCT Number: NCT00712348

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) enzyme replacement therapy.

Completed

Looking for future studies?

Notify Me

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

2 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 is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) ERT. Eligible patients will enter a 12-week Stability Evaluation Period to establish the stability of their disease. Patients with stable disease will then be switched from their imiglucerase treatment to receive intravenous (IV) infusions of taliglucerase alfa every two weeks for a total of 20 IV infusions. The dose of taliglucerase alfa will be equal to each patient's previous imiglucerase dose. The infusions will be administered at the selected investigational site (clinic/hospital), infusion center, or at home. 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.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Males and females, 2 years or older
  • Confirmed diagnosis of Gaucher disease by the enzymatic activity assay
  • Stable Gaucher disease
  • Treatment with imiglucerase (Cerezyme®) for at least 2 years and on a stable maintenance regimen (dose and regimen unchanged, except for situation of drug shortage) for at least the last six months
  • Able to provide written informed consent

Exclusion criteria

  • Currently taking another experimental drug for any condition
  • History of allergy to carrots
  • History of allergy to beta lactam antibiotics
  • Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions
  • Presence of HIV and/or HBsAg and/or hepatitis C infection
  • Presence of unresolved anemia due to iron, folic acid or vitamin B12 deficiency
  • Presence of any significant comorbidity that could confound the interpretation of the clinical response to taliglucerase alfa
  • 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 glucocerebrosidase (prGCD)

Primary outcomes

  1. Hemoglobin

    Time frame: Every 3 months from Baseline to Month 9

Other outcomes

  1. Platelet Count

    Time frame: Every 3 months from Baseline to Month 9

  2. Spleen Volume

    Time frame: Baseline and 9 Months

    Spleen volume measured by MRI in mL

  3. Liver Volume

    Time frame: Baseline and 9 months

    Liver volume measured by MRI

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study title

A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase in Patients With Gaucher Disease Treated With Imiglucerase

Important dates

Study start
2008
Primary completion
2013
Study completion
2013
First posted
Jul 10, 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.

Related clinical trials

Published trials that share one or more normalized conditions with this study.