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Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

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ClinicalTrials.gov Identifier: NCT00712348
Recruitment Status : Completed
First Posted : July 10, 2008
Results First Posted : June 5, 2014
Last Update Posted : October 4, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Gaucher Disease
Intervention Drug: Taliglucerase alfa
Enrollment 31
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Taliglucerase Alfa
Hide Arm/Group Description

Open label taliglucerase alfa treatment

Taliglucerase alfa: Intravenous infusion every 2 weeks

Period Title: Overall Study
Started 31
Completed 30
Not Completed 1
Reason Not Completed
Withdrawal by Subject             1
Arm/Group Title Taliglucerase Alfa
Hide Arm/Group Description

Open label taliglucerase alfa treatment

Taliglucerase alfa: Intravenous infusion every 2 weeks

Overall Number of Baseline Participants 31
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants
<=18 years
5
  16.1%
Between 18 and 65 years
25
  80.6%
>=65 years
1
   3.2%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 31 participants
41.6  (17.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants
Female
14
  45.2%
Male
17
  54.8%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
1
   3.2%
Black or African American
0
   0.0%
White
30
  96.8%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 31 participants
United States 7
Canada 1
Spain 4
Australia 3
Israel 9
United Kingdom 2
Serbia 4
Singapore 1
Religion  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 31 participants
JEWISH - ASHKENAZI 14
JEWISH - NONASHKENAZI 0
NON JEWISH 15
UNREPORTED 2
1.Primary Outcome
Title Hemoglobin
Hide Description [Not Specified]
Time Frame Every 3 months from Baseline to Month 9
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Taliglucerase Alfa
Hide Arm/Group Description:

Open label taliglucerase alfa treatment

Taliglucerase alfa: Intravenous infusion every 2 weeks

Overall Number of Participants Analyzed 30
Mean (Standard Deviation)
Unit of Measure: g/dL
Baseline 13.5  (1.4)
Month 3 13.3  (1.5)
Month 6 13.3  (1.6)
Month 9 13.4  (1.5)
2.Other Pre-specified Outcome
Title Platelet Count
Hide Description [Not Specified]
Time Frame Every 3 months from Baseline to Month 9
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Taliglucerase Alfa
Hide Arm/Group Description:

Open label taliglucerase alfa treatment

Taliglucerase alfa: Intravenous infusion every 2 weeks

Overall Number of Participants Analyzed 30
Mean (Standard Deviation)
Unit of Measure: platelets/mm^3
Baseline 161137  (73387)
Month 3 150800  (66038)
Month 6 157586  (78357)
Month 9 161167  (80820)
3.Other Pre-specified Outcome
Title Spleen Volume
Hide Description Spleen volume measured by MRI in mL
Time Frame Baseline and 9 Months
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Hide Analysis Population Description
25 patients had spleen volume measured by MRI, 2 were MRI phobic and 3 were splenectomized
Arm/Group Title Taliglucerase Alfa
Hide Arm/Group Description:

Open label taliglucerase alfa treatment

Taliglucerase alfa: Intravenous infusion every 2 weeks

Overall Number of Participants Analyzed 25
Mean (Standard Deviation)
Unit of Measure: mL
Baseline 720.5  (579.5)
Month 9 654.7  (535.9)
4.Other Pre-specified Outcome
Title Liver Volume
Hide Description Liver volume measured by MRI
Time Frame Baseline and 9 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
2 patients were MRI phobic
Arm/Group Title Taliglucerase Alfa
Hide Arm/Group Description:

Open label taliglucerase alfa treatment

Taliglucerase alfa: Intravenous infusion every 2 weeks

Overall Number of Participants Analyzed 28
Mean (Standard Deviation)
Unit of Measure: mL
Baseline 1766  (474.5)
Month 9 1716  (455.6)
Time Frame 9 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Taliglucerase Alfa
Hide Arm/Group Description

Open label taliglucerase alfa treatment

Taliglucerase alfa: Intravenous infusion every 2 weeks

All-Cause Mortality
Taliglucerase Alfa
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Taliglucerase Alfa
Affected / at Risk (%) # Events
Total   3/31 (9.68%)    
Renal and urinary disorders   
NEPHROLITHIASIS  1 [1]  1/31 (3.23%)  1
Reproductive system and breast disorders   
PELVIC PROLAPSE  1 [2]  1/31 (3.23%)  1
Respiratory, thoracic and mediastinal disorders   
EPISTAXIS  1 [3]  1/31 (3.23%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
[1]
Not Related
[2]
Prolapsed rectum, cervix and bladder - Not Related
[3]
Right nostril due to perforated septum - Not Related
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Taliglucerase Alfa
Affected / at Risk (%) # Events
Total   29/31 (93.55%)    
Gastrointestinal disorders   
DIARRHOEA  1  2/31 (6.45%)  2
General disorders   
PAIN  1  2/31 (6.45%)  2
Infections and infestations   
NASOPHARYNGITIS  1  4/31 (12.90%)  4
UPPER RESPIRATORY TRACT INFECTION  1  3/31 (9.68%)  3
URINARY TRACT INFECTION  1  3/31 (9.68%)  3
Injury, poisoning and procedural complications   
INFUSION RELATED REACTION  1  4/31 (12.90%)  4
Musculoskeletal and connective tissue disorders   
ARTHRALGIA  1  4/31 (12.90%)  4
PAIN IN EXTREMITY  1  3/31 (9.68%)  3
Nervous system disorders   
HEADACHE  1  4/31 (12.90%)  4
Respiratory, thoracic and mediastinal disorders   
COUGH  1  3/31 (9.68%)  3
Skin and subcutaneous tissue disorders   
PRURITUS  1  2/31 (6.45%)  2
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: VP Medical Affairs
Organization: Protalix Ltd.
Phone: +972-4-9028100
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00712348     History of Changes
Other Study ID Numbers: PB-06-002
First Submitted: July 7, 2008
First Posted: July 10, 2008
Results First Submitted: April 30, 2014
Results First Posted: June 5, 2014
Last Update Posted: October 4, 2018