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

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01422187
First received: August 22, 2011
Last updated: September 21, 2012
Last verified: September 2012
History of Changes
  Purpose

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 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:

U.S. FDA Resources

Further study details as provided by Protalix:

Primary Outcome Measures:
  • Change in spleen volume [ Time Frame: 21 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in liver volume [ Time Frame: 21 months ] [ Designated as safety issue: No ]
  • Change in platelet count [ Time Frame: 21 months ] [ Designated as safety issue: No ]
  • Change in hemoglobin [ Time Frame: 21 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 25
Study Start Date: August 2011
Estimated Study Completion Date: May 2013
Estimated Primary Completion Date: May 2013 (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

  Eligibility

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

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

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

No publications provided

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01422187     History of Changes
Other Study ID Numbers: PB-06-007
Study First Received: August 22, 2011
Last Updated: September 21, 2012
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
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on May 22, 2013