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

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ClinicalTrials.gov Identifier: NCT01132690
Recruitment Status : Completed
First Posted : May 28, 2010
Results First Posted : November 11, 2014
Last Update Posted : October 5, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Gaucher Disease
Intervention Drug: Taliglucerase alfa
Enrollment 11
Recruitment Details  
Pre-assignment Details  
Arm/Group Title 30 Units/kg 60 Units/kg
Hide Arm/Group Description Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Period Title: Overall Study
Started 6 5
Completed 6 5
Not Completed 0 0
Arm/Group Title 30 Units/kg 60 Units/kg Total
Hide Arm/Group Description Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months Total of all reporting groups
Overall Number of Baseline Participants 6 5 11
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 5 participants 11 participants
9.5  (4.0) 6.6  (3.1) 8.2  (3.8)
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 5 participants 11 participants
<=18 years
6
 100.0%
5
 100.0%
11
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 5 participants 11 participants
Female
2
  33.3%
1
  20.0%
3
  27.3%
Male
4
  66.7%
4
  80.0%
8
  72.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 5 participants 11 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
6
 100.0%
4
  80.0%
10
  90.9%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
1
  20.0%
1
   9.1%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 5 participants 11 participants
Hispanic or Latino
3
  50.0%
1
  20.0%
4
  36.4%
Not Hispanic or Latino
3
  50.0%
4
  80.0%
7
  63.6%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants 5 participants 11 participants
Paraguay 3 1 4
South Africa 1 2 3
Israel 2 2 4
Religion  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants 5 participants 11 participants
Jewish - Ashkenazi 0 2 2
Jewish - Non-Ashkenazi 0 0 0
Non Jewish 6 3 9
1.Primary Outcome
Title Hemoglobin
Hide Description median and interquartile range for change from baseline in haemoglobin
Time Frame Every 3 months for 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 30 Units/kg 60 Units/kg
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Overall Number of Participants Analyzed 6 5
Median (Inter-Quartile Range)
Unit of Measure: g/dL
Month 3
1.2
(-0.3 to 1.6)
0.4
(-0.1 to 1.1)
Month 6
1.1
(0.3 to 2.3)
1.5
(1.0 to 1.7)
Month 9
0.9
(0.5 to 1.9)
1.5
(1.4 to 1.6)
Month 12
1.4
(0.2 to 2.5)
1.6
(1.4 to 2.0)
2.Secondary Outcome
Title Chitotriosidase
Hide Description Percent change from baseline in chitotriosidase
Time Frame Every 3 months for 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 30 Units/kg 60 Units/kg
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Overall Number of Participants Analyzed 6 5
Mean (Standard Deviation)
Unit of Measure: Percent Change from Baseline
Month 3 -32.4  (23.5) -32.7  (15.1)
Month 6 -49.6  (19.8) -50.2  (23.7)
Month 9 -57.2  (18.7) -58.9  (21.8)
Month 12 -58.5  (17.5) -66.1  (20.1)
3.Secondary Outcome
Title Spleen Volume
Hide Description Spleen volume measured by MRI
Time Frame Baseline and Month 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 30 Units/kg 60 Units/kg
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Overall Number of Participants Analyzed 6 5
Mean (Standard Deviation)
Unit of Measure: mL
Baseline 1218  (638.4) 1023  (753.4)
Month 12 811.6  (409.6) 524.0  (281.1)
4.Secondary Outcome
Title Platelet Count
Hide Description Mean and standard deviation of platelet count per cubic mm
Time Frame Baseline and 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 30 Units/kg 60 Units/kg
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Overall Number of Participants Analyzed 6 5
Mean (Standard Deviation)
Unit of Measure: platelets per cubic mm
Baseline 162667  (71838) 99600  (42899)
Month 12 208167  (90747) 172200  (89290)
5.Secondary Outcome
Title Chemokine (C-C Motif) Ligand 18 (CCL18)
Hide Description Percent change from baseline in CCL18
Time Frame Every 3 months for 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 30 Units/kg 60 Units/kg
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Overall Number of Participants Analyzed 6 5
Mean (Standard Deviation)
Unit of Measure: Percent Change from Baseline
Month 3 -31.4  (15.3) -18.3  (16.3)
Month 6 -45.7  (11.9) -37.3  (28.8)
Month 9 -49.9  (13.0) -46.5  (22.4)
Month 12 -50.6  (19.4) -52.6  (22.5)
6.Secondary Outcome
Title Liver Volume
Hide Description Liver volume measured by MRI
Time Frame Baseline and Month 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 30 Units/kg 60 Units/kg
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
Overall Number of Participants Analyzed 6 5
Mean (Standard Deviation)
Unit of Measure: mL
Baseline 1214  (424.7) 991.7  (301.3)
Month 12 1116  (366.9) 849.1  (271.9)
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 30 Units/kg 60 Units/kg
Hide Arm/Group Description Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 12 months
All-Cause Mortality
30 Units/kg 60 Units/kg
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
30 Units/kg 60 Units/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/6 (0.00%)      1/5 (20.00%)    
Gastrointestinal disorders     
GASTROINTESTINAL INFLAMMATION * 1  0/6 (0.00%)  0 1/5 (20.00%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (15.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
30 Units/kg 60 Units/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/6 (33.33%)      2/5 (40.00%)    
Gastrointestinal disorders     
VOMITING * 1  2/6 (33.33%)  2 2/5 (40.00%)  2
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (15.0)
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
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01132690     History of Changes
Other Study ID Numbers: PB-06-005
First Submitted: May 26, 2010
First Posted: May 28, 2010
Results First Submitted: October 28, 2014
Results First Posted: November 11, 2014
Last Update Posted: October 5, 2018