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Trial record 1 of 1 for:    NCT00705939
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Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

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ClinicalTrials.gov Identifier: NCT00705939
Recruitment Status : Completed
First Posted : June 27, 2008
Results First Posted : July 15, 2014
Last Update Posted : October 4, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Gaucher Disease
Intervention Drug: Taliglucerase alfa
Enrollment 45
Recruitment Details Patients completing Studies PB-06-001 (NCT00376168) or PB-06-002 (NCT00712348) were offered continued treatment in this study.
Pre-assignment Details  
Arm/Group Title Naive 30 Units/kg Naive 60 Units/kg Switchover
Hide Arm/Group Description Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg Patients previously naive to enzyme replacement therapy from Study PB-06-001 treated with taliglucerase alfa 60 units/kg Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose
Period Title: Overall Study
Started 12 14 18 [1]
Completed 12 12 14
Not Completed 0 2 4
Reason Not Completed
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.
Arm/Group Title Naive 30 Units/kg Naive 60 Units/kg Switchover Total
Hide Arm/Group Description Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg Patients previously naive to enzyme replacement therapy from Study PB-06-001 treated with taliglucerase alfa 60 units/kg Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose Total of all reporting groups
Overall Number of Baseline Participants 12 14 18 44
Hide Baseline Analysis Population Description
All treated patients are included in the analysis
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 12 participants 14 participants 18 participants 44 participants
39.7  (11.8) 36.6  (12.0) 46.5  (13.7) 41.6  (13.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 14 participants 18 participants 44 participants
Female
5
  41.7%
8
  57.1%
9
  50.0%
22
  50.0%
Male
7
  58.3%
6
  42.9%
9
  50.0%
22
  50.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants 14 participants 18 participants 44 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%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
12
 100.0%
13
  92.9%
18
 100.0%
43
  97.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
1
   7.1%
0
   0.0%
1
   2.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 12 participants 14 participants 18 participants 44 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
1.Primary Outcome
Title Spleen Volume
Hide Description Spleen volume measured by MRI
Time Frame Spleen Volume at Baseline and Months 12, 24, and 36
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat. In the Switchover group, two patients did not have MRI and one patient was splenectomized.
Arm/Group Title Naive 30 Units/kg Naive 60 Units/kg Switchover
Hide Arm/Group Description:
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 60 units/kg
Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose.
Overall Number of Participants Analyzed 12 14 15
Mean (Standard Deviation)
Unit of Measure: mL
Baseline 2324.0  (1209.0) 2120.0  (1426.5) 778.0  (666.3)
Month 12 1707.7  (1069.5) 1267.9  (1114.1) 883.7  (760.0)
Month 24 1420.4  (852.3) 946.7  (699.6) 609.1  (442.7)
Month 36 1237.2  (695.7) 761.0  (556.0) 548.2  (433.7)
2.Secondary Outcome
Title Liver Volume
Hide Description Liver volume measured by MRI
Time Frame Liver volume at Baseline and Months 12, 24 and 36
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat. In the Switchover group, two patients did not have MRI .
Arm/Group Title Naive 30 Units/kg Naive 60 Units/kg Switchover
Hide Arm/Group Description:
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 60 units/kg
Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose.
Overall Number of Participants Analyzed 12 14 16
Mean (Standard Deviation)
Unit of Measure: mL
Baseline 2999.7  (779.4) 2470.5  (484.9) 1775.7  (434.4)
Month 12 2515.6  (642.1) 2118.7  (318.1) 1788.9  (378.0)
Month 24 2362.8  (518.7) 1998.2  (291.9) 1757.1  (357.0)
Month 36 2341.1  (553.4) 1971.8  (404.7) 1821.2  (523.6)
3.Secondary Outcome
Title Hemoglobin
Hide Description [Not Specified]
Time Frame Hemoglobin at Baseline and Months 12, 24 and 36
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Naive 30 Units/kg Naive 60 Units/kg Switchover
Hide Arm/Group Description:
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 60 units/kg
Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose
Overall Number of Participants Analyzed 12 14 18
Mean (Standard Deviation)
Unit of Measure: mg/dL
Baseline 12.5  (1.8) 11.4  (2.7) 13.6  (1.6)
Month 12 14.2  (1.7) 13.6  (2.6) 13.6  (1.7)
Month 24 13.8  (1.6) 13.8  (1.8) 13.5  (1.2)
Month 36 14.3  (1.5) 14.1  (2.2) 13.3  (1.2)
4.Secondary Outcome
Title Platelet Count
Hide Description [Not Specified]
Time Frame Platelet count at Baseline and Months 12, 24 and 36
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Naive 30 Units/kg Naive 60 Units/kg Switchover
Hide Arm/Group Description:
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 60 units/kg
Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose
Overall Number of Participants Analyzed 12 14 18
Mean (Standard Deviation)
Unit of Measure: Platelets per cubic millimeter
Baseline 64900  (30133) 69043  (28242) 163833  (88882)
Month 12 80325  (41806) 122857  (53857) 145250  (97426)
Month 24 93333  (53328) 141071  (73896) 174200  (100483)
Month 36 94683  (47526) 144417  (55190) 172467  (89340)
5.Other Pre-specified Outcome
Title Spleen Volume Multiples of Normal (MN)
Hide Description Spleen volume measured by MRI. Normal spleen volume is 2 mL/kg × body weight (kg)
Time Frame Baseline and Months 12, 24, and 36
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat. In the Switchover group, two patients did not have MRI and one patient was splenectomized.
Arm/Group Title Naive 30 Units/kg Naive 60 Units/kg Switchover
Hide Arm/Group Description:
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 60 units/kg
Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose.
Overall Number of Participants Analyzed 12 14 15
Mean (Standard Deviation)
Unit of Measure: Multiples of Normal Spleen Volume
Baseline 16.4  (8.3) 16.8  (14.2) 5.5  (5.4)
Month 12 11.7  (7.2) 9.3  (9.4) 7.0  (6.8)
Month 24 9.6  (1.6) 6.6  (5.3) 4.0  (2.7)
Month 36 8.2  (4.4) 5.6  (4.5) 3.7  (2.9)
6.Other Pre-specified Outcome
Title Liver Volume Multiples of Normal (MN)
Hide Description Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg).
Time Frame Baseline and Months 12, 24 and 36
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to treat. In the Switchover group, two patients did not have MRI.
Arm/Group Title Naive 30 Units/kg Naive 60 Units/kg Switchover
Hide Arm/Group Description:
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg
Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 60 units/kg
Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose.
Overall Number of Participants Analyzed 12 14 16
Mean (Standard Deviation)
Unit of Measure: Multiples of Normal Liver Volume
Baseline 1.7  (0.4) 1.5  (0.4) 1.0  (0.1)
Month 12 1.4  (0.3) 1.2  (0.2) 1.1  (0.2)
Month 24 1.3  (0.2) 1.1  (0.2) 0.9  (0.2)
Month 36 1.3  (0.2) 1.1  (0.2) 1.0  (0.3)
Time Frame 36 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Naive 30 Units/kg Naive 60 Units/kg Switchover
Hide Arm/Group Description Patients previously naive to enzyme replacement therapy treated in Study PB-06-001 with taliglucerase alfa 30 units/kg Patients previously naive to enzyme replacement therapy from Study PB-06-001 treated with taliglucerase alfa 60 units/kg Patients previously treated with imiglucerase in Study PB-06-002 treated with taliglucerase alfa at the same dose as their previous imiglucerase dose
All-Cause Mortality
Naive 30 Units/kg Naive 60 Units/kg Switchover
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Naive 30 Units/kg Naive 60 Units/kg Switchover
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/12 (16.67%)      2/14 (14.29%)      3/18 (16.67%)    
Blood and lymphatic system disorders       
AUTOIMMUNE THROMBOCYTOPENIA  1 [1]  0/12 (0.00%)  0 1/14 (7.14%)  1 0/18 (0.00%)  0
Injury, poisoning and procedural complications       
HEAD INJURY  1 [2]  0/12 (0.00%)  0 1/14 (7.14%)  1 0/18 (0.00%)  0
PNEUMOTHORAX TRAUMATIC  1 [1]  0/12 (0.00%)  0 0/14 (0.00%)  0 1/18 (5.56%)  1
Musculoskeletal and connective tissue disorders       
ARTHRALGIA  1 [3]  0/12 (0.00%)  0 0/14 (0.00%)  0 1/18 (5.56%)  1
OSTEONECROSIS  1 [4]  0/12 (0.00%)  0 1/14 (7.14%)  1 0/18 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
HAEMANGIOMA  1 [5]  1/12 (8.33%)  1 0/14 (0.00%)  0 0/18 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
PULMONARY EMBOLISM  1 [1]  1/12 (8.33%)  1 0/14 (0.00%)  0 0/18 (0.00%)  0
Surgical and medical procedures       
RENAL STONE REMOVAL  1 [1]  0/12 (0.00%)  0 0/14 (0.00%)  0 1/18 (5.56%)  1
TONSILLECTOMY  1 [1]  1/12 (8.33%)  1 0/14 (0.00%)  0 0/18 (0.00%)  0
TOOTH EXTRACTION  1 [1]  1/12 (8.33%)  1 0/14 (0.00%)  0 0/18 (0.00%)  0
VOCAL CORD POLYPECTOMY  1 [1]  1/12 (8.33%)  1 0/14 (0.00%)  0 0/18 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
[1]
Not Related
[2]
Head trauma - Not Related
[3]
Surgery: Left knee arthroscopy - Not Related
[4]
Right acetabular necrosis - Not Related
[5]
pain due to hemangioma left knee - Not Related
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Naive 30 Units/kg Naive 60 Units/kg Switchover
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   12/12 (100.00%)      12/14 (85.71%)      17/18 (94.44%)    
Gastrointestinal disorders       
DIARRHOEA  1  3/12 (25.00%)  3 0/14 (0.00%)  0 3/18 (16.67%)  3
VOMITING  1  1/12 (8.33%)  1 1/14 (7.14%)  1 3/18 (16.67%)  3
General disorders       
FATIGUE  1  2/12 (16.67%)  2 2/14 (14.29%)  2 2/18 (11.11%)  2
PAIN  1  2/12 (16.67%)  2 1/14 (7.14%)  1 1/18 (5.56%)  1
PYREXIA  1  0/12 (0.00%)  0 3/14 (21.43%)  3 4/18 (22.22%)  4
Infections and infestations       
GASTROENTERITIS  1  2/12 (16.67%)  2 1/14 (7.14%)  1 1/18 (5.56%)  1
NASOPHARYNGITIS  1  2/12 (16.67%)  2 3/14 (21.43%)  3 7/18 (38.89%)  7
PHARYNGITIS  1  2/12 (16.67%)  2 0/14 (0.00%)  0 1/18 (5.56%)  1
RESPIRATORY TRACT INFECTION  1  0/12 (0.00%)  0 0/14 (0.00%)  0 2/18 (11.11%)  2
SINUSITIS  1  0/12 (0.00%)  0 3/14 (21.43%)  3 2/18 (11.11%)  2
UPPER RESPIRATORY TRACT INFECTION  1  3/12 (25.00%)  3 3/14 (21.43%)  3 3/18 (16.67%)  3
URINARY TRACT INFECTION  1  0/12 (0.00%)  0 0/14 (0.00%)  0 2/18 (11.11%)  2
Injury, poisoning and procedural complications       
LIMB INJURY  1  1/12 (8.33%)  1 2/14 (14.29%)  2 0/18 (0.00%)  0
Musculoskeletal and connective tissue disorders       
ARTHRALGIA  1  4/12 (33.33%)  4 4/14 (28.57%)  4 4/18 (22.22%)  4
BACK PAIN  1  2/12 (16.67%)  2 2/14 (14.29%)  2 2/18 (11.11%)  2
MUSCULOSKELETAL PAIN  1  0/12 (0.00%)  0 2/14 (14.29%)  2 3/18 (16.67%)  3
PAIN IN EXTREMITY  1  2/12 (16.67%)  2 4/14 (28.57%)  4 2/18 (11.11%)  2
Nervous system disorders       
HEADACHE  1  4/12 (33.33%)  4 3/14 (21.43%)  3 2/18 (11.11%)  2
Respiratory, thoracic and mediastinal disorders       
COUGH  1  0/12 (0.00%)  0 1/14 (7.14%)  1 3/18 (16.67%)  3
EPISTAXIS  1  2/12 (16.67%)  2 1/14 (7.14%)  1 0/18 (0.00%)  0
OROPHARYNGEAL PAIN  1  1/12 (8.33%)  1 2/14 (14.29%)  2 1/18 (5.56%)  1
Skin and subcutaneous tissue disorders       
ERYTHEMA  1  3/12 (25.00%)  3 0/14 (0.00%)  0 0/18 (0.00%)  0
PRURITUS  1  1/12 (8.33%)  1 2/14 (14.29%)  2 1/18 (5.56%)  1
Surgical and medical procedures       
ENDODONTIC PROCEDURE  1  0/12 (0.00%)  0 2/14 (14.29%)  2 0/18 (0.00%)  0
Vascular disorders       
HYPERTENSION  1  2/12 (16.67%)  2 3/14 (21.43%)  3 1/18 (5.56%)  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: Vice President Product Development
Organization: Protalix Ltd.
Phone: +(972) 4-9889488 ext 137
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00705939     History of Changes
Other Study ID Numbers: PB-06-003
First Submitted: June 25, 2008
First Posted: June 27, 2008
Results First Submitted: April 30, 2014
Results First Posted: July 15, 2014
Last Update Posted: October 4, 2018