Skip to main content
OpenTrials
Completed

NCT Number: NCT01411228

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

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).

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

2 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Shaare Zedek Medical Center, Jerusalem, Israel

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Taliglucerase alfa

Drug

Taliglucerase alfa for infusion every two weeks for 24 months

Other names: prGCD, plant cell expressed glucocerebrosidase

Primary outcomes

  1. 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 outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Important dates

Study start
2011
Primary completion
2014
Study completion
2014
First posted
Aug 8, 2011
Registry last updated
Sep 7, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.