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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: 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 Summary:
Sponsor: Protalix

Current Primary Outcome: 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: Hemoglobin [ Time Frame: Every 3 months for 2 years ]

median percentage and the interquartile range for change from baseline in haemoglobin


Current Secondary Outcome:

  • 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:

  • 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


Information By: Protalix

Dates:
Date Received: August 4, 2011
Date Started: September 2011
Date Completion:
Last Updated: June 20, 2016
Last Verified: June 2016