Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT00712348
First received: July 7, 2008
Last updated: May 30, 2014
Last verified: May 2014

July 7, 2008
May 30, 2014
December 2008
April 2013   (final data collection date for primary outcome measure)
Hemoglobin [ Time Frame: Every 3 months from Baseline to Month 9 ] [ Designated as safety issue: No ]
Adverse events [ Time Frame: Continuous ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT00712348 on ClinicalTrials.gov Archive Site
Not Provided
Platelet count [ Time Frame: Monthly ] [ Designated as safety issue: Yes ]
  • Platelet Count [ Time Frame: Every 3 months from Baseline to Month 9 ] [ Designated as safety issue: No ]
  • Spleen Volume [ Time Frame: Baseline and 9 Months ] [ Designated as safety issue: No ]
    Spleen volume measured by MRI in mL
  • Liver Volume [ Time Frame: Baseline and 9 months ] [ Designated as safety issue: No ]
    Liver volume measured by MRI
Not Provided
 
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
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

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.

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.

Interventional
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Gaucher Disease
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Name: Plant cell expressed recombinant glucocerebrosidase (prGCD)
Experimental: Taliglucerase alfa
Open label taliglucerase alfa treatment
Intervention: Drug: Taliglucerase alfa
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
31
May 2013
April 2013   (final data collection date for primary outcome measure)

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
Both
2 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Australia,   Canada,   Israel,   Spain,   United Kingdom
 
NCT00712348
PB-06-002
Yes
Protalix
Protalix
Not Provided
Principal Investigator: Ari Zimran, MD Shaare Zedek Medical Center, Jerusalem
Protalix
May 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP