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