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A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01132690
First received: May 26, 2010
Last updated: November 10, 2014
Last verified: November 2014
Results First Received: October 28, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   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
No text entered.

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
30 Units/kg Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
60 Units/kg Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months

Participant Flow:   Overall Study
    30 Units/kg     60 Units/kg  
STARTED     6     5  
COMPLETED     6     5  
NOT COMPLETED     0     0  



  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.
No text entered.

Reporting Groups
  Description
30 Units/kg Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
60 Units/kg Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Total Total of all reporting groups

Baseline Measures
    30 Units/kg     60 Units/kg     Total  
Number of Participants  
[units: participants]
  6     5     11  
Age  
[units: years]
Mean ± Standard Deviation
  9.5  ± 4.0     6.6  ± 3.1     8.2  ± 3.8  
Age  
[units: participants]
     
<=18 years     6     5     11  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Gender  
[units: participants]
     
Female     2     1     3  
Male     4     4     8  
Race (NIH/OMB)  
[units: participants]
     
American Indian or Alaska Native     0     0     0  
Asian     0     0     0  
Native Hawaiian or Other Pacific Islander     0     0     0  
Black or African American     0     0     0  
White     6     4     10  
More than one race     0     0     0  
Unknown or Not Reported     0     1     1  
Ethnicity (NIH/OMB)  
[units: participants]
     
Hispanic or Latino     3     1     4  
Not Hispanic or Latino     3     4     7  
Unknown or Not Reported     0     0     0  
Region of Enrollment  
[units: participants]
     
Paraguay     3     1     4  
South Africa     1     2     3  
Israel     2     2     4  
Religion  
[units: participants]
     
Jewish - Ashkenazi     0     2     2  
Jewish - Non-Ashkenazi     0     0     0  
Non Jewish     6     3     9  



  Outcome Measures
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1.  Primary:   Hemoglobin   [ Time Frame: Every 3 months for 12 months ]

2.  Secondary:   Chitotriosidase   [ Time Frame: Every 3 months for 12 months ]

3.  Secondary:   Spleen Volume   [ Time Frame: Baseline and Month 12 ]

4.  Secondary:   Platelet Count   [ Time Frame: Baseline and 12 months ]

5.  Secondary:   Chemokine (C-C Motif) Ligand 18 (CCL18)   [ Time Frame: Every 3 months for 12 months ]

6.  Secondary:   Liver Volume   [ Time Frame: Baseline and Month 12 ]


  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: Senior VP Development
Organization: Protalix Biotherapeutics
phone: +972-4-902-8100
e-mail: einat@protalix.com


No publications provided


Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01132690     History of Changes
Other Study ID Numbers: PB-06-005
Study First Received: May 26, 2010
Results First Received: October 28, 2014
Last Updated: November 10, 2014
Health Authority: United States: Food and Drug Administration
Israel: Ministry of Health
Canada: Health Canada