Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT00705939
First received: June 25, 2008
Last updated: July 14, 2014
Last verified: July 2014

June 25, 2008
July 14, 2014
June 2008
May 2012   (final data collection date for primary outcome measure)
Spleen Volume [ Time Frame: Spleen Volume at Baseline and Months 12, 24, and 36 ] [ Designated as safety issue: No ]
Spleen volume measured by MRI
Spleen Volume [ Time Frame: 3, 9, and 15 months ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00705939 on ClinicalTrials.gov Archive Site
  • Liver Volume [ Time Frame: Liver volume at Baseline and Months 12, 24 and 36 ] [ Designated as safety issue: No ]
    Liver volume measured by MRI
  • Hemoglobin [ Time Frame: Hemoglobin at Baseline and Months 12, 24 and 36 ] [ Designated as safety issue: No ]
  • Platelet Count [ Time Frame: Platelet count at Baseline and Months 12, 24 and 36 ] [ Designated as safety issue: No ]
Liver Volume [ Time Frame: 3, 9, and 15 months ] [ Designated as safety issue: No ]
  • Spleen Volume Multiples of Normal (MN) [ Time Frame: Baseline and Months 12, 24, and 36 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg).
Not Provided
 
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) 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 an extension trial to Study NCT00376168 and NCT00712348.

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.

Interventional
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Gaucher Disease
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Names:
  • Plant Cell Expressed Recombinant Human Glucocerebrosidase
  • prGCD
  • 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
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
45
August 2013
May 2012   (final data collection date for primary outcome measure)

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
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Australia,   Canada,   Chile,   Israel,   South Africa,   Spain,   United Kingdom
 
NCT00705939
PB-06-003
Yes
Protalix
Protalix
Not Provided
Principal Investigator: Ari Zimran, MD Shaare Zedek Medical Center, Jerusalem, Israel
Protalix
July 2014

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