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Trial record 1 of 1 for:    NCT00705939
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Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

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ClinicalTrials.gov Identifier: NCT00705939
Recruitment Status : Completed
First Posted : June 27, 2008
Results First Posted : July 15, 2014
Last Update Posted : October 4, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE June 25, 2008
First Posted Date  ICMJE June 27, 2008
Results First Submitted Date  ICMJE April 30, 2014
Results First Posted Date  ICMJE July 15, 2014
Last Update Posted Date October 4, 2018
Study Start Date  ICMJE June 2008
Actual Primary Completion Date May 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 14, 2014)
Spleen Volume [ Time Frame: Spleen Volume at Baseline and Months 12, 24, and 36 ]
Spleen volume measured by MRI
Original Primary Outcome Measures  ICMJE
 (submitted: June 25, 2008)
Spleen Volume [ Time Frame: 3, 9, and 15 months ]
Change History Complete list of historical versions of study NCT00705939 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 14, 2014)
  • Liver Volume [ Time Frame: Liver volume at Baseline and Months 12, 24 and 36 ]
    Liver volume measured by MRI
  • Hemoglobin [ Time Frame: Hemoglobin at Baseline and Months 12, 24 and 36 ]
  • Platelet Count [ Time Frame: Platelet count at Baseline and Months 12, 24 and 36 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: June 25, 2008)
Liver Volume [ Time Frame: 3, 9, and 15 months ]
Current Other Pre-specified Outcome Measures
 (submitted: July 14, 2014)
  • 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)
  • 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).
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Official Title  ICMJE A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
Brief Summary

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.

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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Gaucher Disease
Intervention  ICMJE Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Names:
  • Plant Cell Expressed Recombinant Human Glucocerebrosidase
  • prGCD
Study Arms  ICMJE
  • Experimental: Naive 30 Units/kg
    Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
    Intervention: Drug: Taliglucerase alfa
  • Experimental: Naive 60 Units/kg
    Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
    Intervention: Drug: Taliglucerase alfa
  • Experimental: Switchover
    Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)
    Intervention: Drug: Taliglucerase alfa
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 14, 2014)
45
Original Estimated Enrollment  ICMJE
 (submitted: June 25, 2008)
25
Actual Study Completion Date  ICMJE August 2013
Actual Primary Completion Date May 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   Chile,   Israel,   South Africa,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00705939
Other Study ID Numbers  ICMJE PB-06-003
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Pfizer
Verification Date September 2018

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