Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Tracking Information
First Submitted Date  ICMJE August 4, 2011
First Posted Date  ICMJE August 8, 2011
Results First Submitted Date  ICMJE November 6, 2015
Results First Posted Date  ICMJE January 18, 2016
Last Update Posted Date September 7, 2018
Study Start Date  ICMJE September 2011
Actual Primary Completion Date July 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 11, 2015)
Hemoglobin [ Time Frame: Baseline, months 9, 12 and 24 ]
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.
Original Primary Outcome Measures  ICMJE
 (submitted: August 4, 2011)
Hemoglobin [ Time Frame: Every 3 months for 2 years ]
median percentage and the interquartile range for change from baseline in haemoglobin
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 11, 2015)
  • Chitotriosidase [ Time Frame: Baseline, months 9, 12 and 24 ]
    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.
  • Spleen Volume [ Time Frame: Baseline, months 12 and 24 ]
    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.
  • Platelet Count [ Time Frame: Baseline, months 9, 12, 24 and 33-36 ]
    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.
  • Liver Volume [ Time Frame: Baseline, months 12 and 24 ]
    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.
Original Secondary Outcome Measures  ICMJE
 (submitted: August 4, 2011)
  • Chitotriosidase [ Time Frame: Every 3 months for 2 years ]
    Percent change from baseline in chitotriosidase
  • Spleen and liver volume [ Time Frame: at 1 and 2 years ]
    Percent change in spleen and liver volume measured by MRI (or ultrasound)
  • Platelet Count [ Time Frame: Every 3 months for 2 years ]
    Percent change from baseline in platelet count
  • Anti taliglucerase alfa antibodies [ Time Frame: Every 3 months for 2 years ]
    Occurrence of positive antibody response
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Official Title  ICMJE A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Brief Summary A protocol to extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols PB-06-002 (switchover study from imiglucerase) or PB-06-005 (naïve treatment with taliglucerase alfa).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Gaucher Disease
Intervention  ICMJE Drug: Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 24 months
Other Name: prGCD, plant cell expressed glucocerebrosidase
Study Arms  ICMJE
  • Experimental: 60 Units/kg
    Intervention: Drug: Taliglucerase alfa
  • Experimental: 30 Units/kg
    Intervention: Drug: Taliglucerase alfa
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 4, 2011)
15
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE August 2014
Actual Primary Completion Date July 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Successful completion of Protocol PB-06-002 or PB-06-005
  • The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent

Exclusion Criteria:

  • Currently taking another investigational drug for any condition.
  • Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
  • Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Israel,   Paraguay,   South Africa
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01411228
Other Study ID Numbers  ICMJE PB-06-006
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Pfizer
Verification Date September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP