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

Overview

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).

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Triple (Participant, Care Provider, Investigator)
  • Study Primary Completion Date: July 2014

Interventions

  • Drug: Taliglucerase alfa
    • Taliglucerase alfa for infusion every two weeks for 24 months

Arms, Groups and Cohorts

  • Experimental: 60 Units/kg
  • Experimental: 30 Units/kg

Clinical Trial Outcome Measures

Primary Measures

  • 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.

Secondary Measures

  • 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.

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 2 Years

Maximum Age: 18 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Pfizer
  • Provider of Information About this Clinical Study
    • Sponsor

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.