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A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

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ClinicalTrials.gov Identifier: NCT01411228
Recruitment Status : Completed
First Posted : August 8, 2011
Results First Posted : January 18, 2016
Last Update Posted : September 7, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Gaucher Disease
Intervention Drug: Taliglucerase alfa
Enrollment 15
Recruitment Details  
Pre-assignment Details  
Arm/Group Title 60 Units/kg 30 Units/kg Switchover
Hide Arm/Group Description Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Period Title: Overall Study
Started 5 5 5
Completed 4 5 4
Not Completed 1 0 1
Reason Not Completed
Lost to Follow-up             0             0             1
Withdrawal by Subject             1             0             0
Arm/Group Title 60 Units/kg 30 Units/kg Dose Adjusted Total
Hide Arm/Group Description Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months Taliglucerase alfa: Dose increased from 30 Units/kg to 45 or 60 Units/kg Total of all reporting groups
Overall Number of Baseline Participants 5 5 5 15
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 5 participants 5 participants 5 participants 15 participants
6.6  (3.1) 8.6  (3.8) 13.0  (4.1) 9.4  (4.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 5 participants 5 participants 15 participants
Female
1
  20.0%
2
  40.0%
2
  40.0%
5
  33.3%
Male
4
  80.0%
3
  60.0%
3
  60.0%
10
  66.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants 5 participants 5 participants 15 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
1
  20.0%
1
   6.7%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
4
  80.0%
5
 100.0%
4
  80.0%
13
  86.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
  20.0%
0
   0.0%
0
   0.0%
1
   6.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 5 participants 5 participants 5 participants 15 participants
South Africa 2 1 0 3
Israel 2 1 0 3
Spain 0 0 2 2
Serbia 0 0 2 2
Paraguay 1 3 0 4
Singapore 0 0 1 1
1.Primary Outcome
Title Hemoglobin
Hide Description Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Time Frame Baseline, months 9, 12 and 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program
Arm/Group Title 60 Units/kg 30 Units/kg Switchover
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Maintained dose from PB-06-002 study
Overall Number of Participants Analyzed 5 5 5
Median (Inter-Quartile Range)
Unit of Measure: g/dL
Baseline
10.6
(9.1 to 12.3)
11.2
(9.1 to 12.3)
13.5
(12.9 to 14.2)
Month 9
11.9
(10.5 to 13.8)
12.5
(10.1 to 13.7)
13.9
(12.5 to 15.2)
Month 12
12.2
(11.2 to 13.7)
12.8
(11.2 to 14.2)
13.7
(12.0 to 15.1)
Month 24
12.8
(11.0 to 14.2)
13.1
(11.5 to 14.5)
14.2
(12.1 to 16.0)
2.Secondary Outcome
Title Chitotriosidase
Hide Description Chitotriosidase. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Time Frame Baseline, months 9, 12 and 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Chitotriosidase was not analyzed for the subjects in the Switchover group
Arm/Group Title 60 Units/kg 30 Units/kg Switchover
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Maintained dose from PB-06-002 study
Overall Number of Participants Analyzed 5 4 0
Mean (Standard Deviation)
Unit of Measure: nmol/mL*h
Baseline 34968  (20928) 30783  (17771)
Month 9 17416  (16821) 13983  (10562)
Month 12 14677  (14418) 13823  (11525)
Month 24 7449.5  (6966.5) 10079  (9452.4)
3.Secondary Outcome
Title Spleen Volume
Hide Description Spleen volume measured by MRI (or ultrasound). Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Time Frame Baseline, months 12 and 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program
Arm/Group Title 60 Units/kg 30 Units/kg Switchover
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Maintained dose from PB-06-002 study
Overall Number of Participants Analyzed 5 5 5
Mean (Standard Error)
Unit of Measure: Milliliters
Baseline 1023.1  (336.9) 1244.7  (317.6) 313.0  (69.8)
12 Months 524.0  (125.7) 759.0  (194.4) 274.1  (39.3)
24 Months 395.8  (76.8) 719.0  (161.2) 249.5  (69.8)
33-36 Months 306.1  (55.1) 673.2  (128.7) 185.4  (12.6)
4.Secondary Outcome
Title Platelet Count
Hide Description Platelet count. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Time Frame Baseline, months 9, 12, 24 and 33-36
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program
Arm/Group Title 60 Units/kg 30 Units/kg Switchover
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Maintained dose from PB-06-002 study
Overall Number of Participants Analyzed 5 5 5
Mean (Standard Error)
Unit of Measure: Platelets/mm^3
Baseline 99600  (19185) 182000  (27009) 164587  (17321)
Month 9 172600  (38667) 221400  (30626) 177400  (16795)
Month 12 172200  (39932) 233800  (32759) 189000  (7733.0)
Month 24 192800  (26673) 207600  (18291) 234800  (14867)
Month 33-36 243750  (18585) 220200  (29231) 210000  (10000)
5.Secondary Outcome
Title Liver Volume
Hide Description Liver volume by MRI or Ultrasound. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Time Frame Baseline, months 12 and 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Two Switchover Patients completed 18 months treatment due to early closure of the study site and continued in a compassionate use program
Arm/Group Title 60 Units/kg 30 Units/kg Switchover
Hide Arm/Group Description:
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months
Taliglucerase alfa: Maintained dose from PB-06-002 study
Overall Number of Participants Analyzed 5 5 5
Mean (Standard Error)
Unit of Measure: Milliliters
Baseline 991.7  (134.7) 1236.5  (210.6) 1345.6  (193.1)
Month 12 849.1  (121.6) 1140.6  (180.9) 1460.7  (227.8)
Month 24 865.5  (82.4) 1254.4  (114.9) 1480.7  (253.3)
Month 33-36 890.6  (118.2) 1244.8  (89.1) 1274.2  (189.7)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 60 Units/kg 30 Units/kg Switchover
Hide Arm/Group Description Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months Taliglucerase alfa: Taliglucerase alfa for infusion every two weeks for 24 months Taliglucerase alfa: Maintained dose from PB-06-002 study
All-Cause Mortality
60 Units/kg 30 Units/kg Switchover
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
60 Units/kg 30 Units/kg Switchover
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/5 (20.00%)      0/5 (0.00%)      0/5 (0.00%)    
Infections and infestations       
Dengue Fever  1 [1]  1/5 (20.00%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
[1]
(Grade 2 Dengue Fever), which required hospitalization, resolved in 5 weeks and was assessed as not related to study drug
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
60 Units/kg 30 Units/kg Switchover
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/5 (100.00%)      4/5 (80.00%)      5/5 (100.00%)    
Blood and lymphatic system disorders       
LYMPHADENOPATHY  1  1/5 (20.00%)  1 2/5 (40.00%)  2 0/5 (0.00%)  0
Ear and labyrinth disorders       
EAR PAIN  1  2/5 (40.00%)  2 0/5 (0.00%)  0 0/5 (0.00%)  0
Gastrointestinal disorders       
ABDOMINAL PAIN  1  1/5 (20.00%)  1 2/5 (40.00%)  2 0/5 (0.00%)  0
DIARRHOEA  1  3/5 (60.00%)  3 0/5 (0.00%)  0 0/5 (0.00%)  0
Infections and infestations       
DENGUE FEVER  1  0/5 (0.00%)  0 2/5 (40.00%)  2 0/5 (0.00%)  0
UPPER RESPIRATORY TRACT INFECTION  1  2/5 (40.00%)  2 1/5 (20.00%)  1 1/5 (20.00%)  1
NASOPHARYNGITIS  1  1/5 (20.00%)  1 0/5 (0.00%)  0 2/5 (40.00%)  2
Musculoskeletal and connective tissue disorders       
PAIN IN EXTREMITY  1  2/5 (40.00%)  2 1/5 (20.00%)  1 0/5 (0.00%)  0
Nervous system disorders       
HEADACHE  1  2/5 (40.00%)  2 1/5 (20.00%)  1 1/5 (20.00%)  1
Respiratory, thoracic and mediastinal disorders       
COUGH  1  2/5 (40.00%)  2 1/5 (20.00%)  1 1/5 (20.00%)  1
Indicates events were collected by 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: Glen Park
Organization: Target Health Inc.
Phone: 212-681-2100
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01411228     History of Changes
Other Study ID Numbers: PB-06-006
First Submitted: August 4, 2011
First Posted: August 8, 2011
Results First Submitted: November 6, 2015
Results First Posted: January 18, 2016
Last Update Posted: September 7, 2018