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
Results First Received: April 30, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Gaucher Disease
Intervention: Drug: Taliglucerase alfa

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patients completing Studies PB-06-001 (NCT00376168) or PB-06-002 (NCT00712348) were offered continued treatment in this study.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Naive 30 Units/kg Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg
Naive 60 Units/kg Patients previously naive to enzyme replacement therapy from Study PB-06-001 treated with taliglucerase alfa 60 units/kg
Switchover Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose

Participant Flow:   Overall Study
    Naive 30 Units/kg     Naive 60 Units/kg     Switchover  
STARTED     12     14     18 [1]
COMPLETED     12     12     14  
NOT COMPLETED     0     2     4  
Protocol Violation                 0                 0                 1  
Withdrawal by Subject                 0                 1                 3  
Physician Decision                 0                 1                 0  
[1] 1 subject enrolled but not treated in this protocol.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
All treated patients are included in the analysis

Reporting Groups
  Description
Naive 30 Units/kg Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg
Naive 60 Units/kg Patients previously naive to enzyme replacement therapy from Study PB-06-001 treated with taliglucerase alfa 60 units/kg
Switchover Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose
Total Total of all reporting groups

Baseline Measures
    Naive 30 Units/kg     Naive 60 Units/kg     Switchover     Total  
Number of Participants  
[units: participants]
  12     14     18     44  
Age  
[units: years]
Mean ± Standard Deviation
  39.7  ± 11.8     36.6  ± 12.0     46.5  ± 13.7     41.6  ± 13.0  
Gender  
[units: participants]
       
Female     5     8     9     22  
Male     7     6     9     22  
Race (NIH/OMB)  
[units: participants]
       
American Indian or Alaska Native     0     0     0     0  
Asian     0     0     0     0  
Native Hawaiian or Other Pacific Islander     0     0     0     0  
Black or African American     0     0     0     0  
White     12     13     18     43  
More than one race     0     0     0     0  
Unknown or Not Reported     0     1     0     1  
Region of Enrollment  
[units: participants]
       
United States     0     0     6     6  
Canada     1     1     1     3  
Spain     0     1     2     3  
Australia     0     0     1     1  
Chile     1     1     0     2  
South Africa     1     1     0     2  
Israel     3     4     6     13  
United Kingdom     0     1     0     1  
Italy     1     1     0     2  
Serbia     2     2     2     6  
Mexico     3     2     0     5  



  Outcome Measures
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1.  Primary:   Spleen Volume   [ Time Frame: Spleen Volume at Baseline and Months 12, 24, and 36 ]

2.  Secondary:   Liver Volume   [ Time Frame: Liver volume at Baseline and Months 12, 24 and 36 ]

3.  Secondary:   Hemoglobin   [ Time Frame: Hemoglobin at Baseline and Months 12, 24 and 36 ]

4.  Secondary:   Platelet Count   [ Time Frame: Platelet count at Baseline and Months 12, 24 and 36 ]

5.  Other Pre-specified:   Spleen Volume Multiples of Normal (MN)   [ Time Frame: Baseline and Months 12, 24, and 36 ]

6.  Other Pre-specified:   Liver Volume Multiples of Normal (MN)   [ Time Frame: Baseline and Months 12, 24 and 36 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Vice President Product Development
Organization: Protalix Ltd.
phone: +(972) 4-9889488 ext 137
e-mail: einata@protalix.com


No publications provided


Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT00705939     History of Changes
Other Study ID Numbers: PB-06-003
Study First Received: June 25, 2008
Results First Received: April 30, 2014
Last Updated: July 14, 2014
Health Authority: United States: Food and Drug Administration
Israel: Ministry of Health