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Trial record 35 of 101 for:    Gaucher Disease

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

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ClinicalTrials.gov Identifier: NCT01422187
Recruitment Status : Completed
First Posted : August 23, 2011
Results First Posted : December 14, 2015
Last Update Posted : May 19, 2016
Sponsor:
Information provided by (Responsible Party):
Protalix

Brief Summary:
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 or disease Intervention/treatment Phase
Gaucher Disease Drug: Taliglucerase alfa Phase 3

Expanded Access : An investigational treatment associated with this study is no longer available outside the clinical trial.   More info ...

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
Study Start Date : August 2011
Actual Primary Completion Date : May 2014
Actual Study Completion Date : September 2014


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Spleen Volume [ Time Frame: 60 months ]
    Spleen volume measured by MRI


Secondary Outcome Measures :
  1. Liver Volume [ Time Frame: 60 months ]
    Liver volume by MRI

  2. Platelet Count [ Time Frame: 60 months ]
    Platelet count measure annually

  3. Hemoglobin [ Time Frame: 60 months ]
    Hemoglobin measure yearly



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01422187


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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01422187     History of Changes
Other Study ID Numbers: PB-06-007
First Posted: August 23, 2011    Key Record Dates
Results First Posted: December 14, 2015
Last Update Posted: May 19, 2016
Last Verified: April 2016

Keywords provided by Protalix:
enzyme replacement therapy

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