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

This study has been completed.
Information provided by (Responsible Party):
ClinicalTrials.gov Identifier:
First received: August 22, 2011
Last updated: November 6, 2015
Last verified: November 2015
This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The duration of treatment will be a maximum of 21 months or until taliglucerase alfa is commercially available to the subject at the discretion of the Sponsor.

Condition Intervention Phase
Gaucher Disease
Drug: Taliglucerase alfa
Phase 3

Access to an investigational treatment associated with this study is no longer available outside the clinical trial.   More info ...

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

Resource links provided by NLM:

Further study details as provided by Protalix:

Primary Outcome Measures:
  • Spleen Volume [ Time Frame: 60 months ] [ Designated as safety issue: No ]
    Spleen volume measured by MRI

Secondary Outcome Measures:
  • Liver Volume [ Time Frame: 60 months ] [ Designated as safety issue: No ]
    Liver volume by MRI

  • Platelet Count [ Time Frame: 60 months ] [ Designated as safety issue: No ]
    Platelet count measure annually

  • Hemoglobin [ Time Frame: 60 months ] [ Designated as safety issue: No ]
    Hemoglobin measure yearly

Enrollment: 19
Study Start Date: August 2011
Study Completion Date: September 2014
Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Taliglucerase alfa 30 units/kg
Subjects randomized to receive 30 units/kg
Drug: Taliglucerase alfa
Taliglucerase infusion every two weeks for 21 months
Experimental: Taliglucerase alfa 60 units/kg
Subjects randomized to 60 units/kg
Drug: Taliglucerase alfa
Taliglucerase infusion every two weeks for 21 months


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Successfully completed Protocol PB-06-001 and enrolled in Protocol PB-06-003
  • The subject signs an informed consent

Exclusion Criteria:

  • Currently taking another investigational drug for any condition.
  • 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01422187

Sponsors and Collaborators
Study Director: Einat Almon, PhD Protalix Ltd
  More Information

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01422187     History of Changes
Other Study ID Numbers: PB-06-007 
Study First Received: August 22, 2011
Results First Received: November 6, 2015
Last Updated: November 6, 2015
Health Authority: United States: Food and Drug Administration
Israel: Ministry of Health

Keywords provided by Protalix:
enzyme replacement therapy

Additional relevant MeSH terms:
Gaucher Disease
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases

ClinicalTrials.gov processed this record on April 27, 2016