search
Back to results

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

Primary Purpose

Gaucher Disease

Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Taliglucerase alfa
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gaucher Disease focused on measuring enzyme replacement therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    Experimental

    Arm Label

    Taliglucerase alfa 30 units/kg

    Taliglucerase alfa 60 units/kg

    Arm Description

    Subjects randomized to receive 30 units/kg

    Subjects randomized to 60 units/kg

    Outcomes

    Primary Outcome Measures

    Spleen Volume
    Spleen volume measured by MRI

    Secondary Outcome Measures

    Liver Volume
    Liver volume by MRI
    Platelet Count
    Platelet count measure annually
    Hemoglobin
    Hemoglobin measure yearly

    Full Information

    First Posted
    August 22, 2011
    Last Updated
    April 18, 2023
    Sponsor
    Pfizer
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT01422187
    Brief Title
    A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
    Official Title
    A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2023
    Overall Recruitment Status
    Completed
    Study Start Date
    August 2011 (undefined)
    Primary Completion Date
    May 2014 (Actual)
    Study Completion Date
    September 2014 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Pfizer

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Gaucher Disease
    Keywords
    enzyme replacement therapy

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Parallel Assignment
    Masking
    None (Open Label)
    Allocation
    Randomized
    Enrollment
    19 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Taliglucerase alfa 30 units/kg
    Arm Type
    Experimental
    Arm Description
    Subjects randomized to receive 30 units/kg
    Arm Title
    Taliglucerase alfa 60 units/kg
    Arm Type
    Experimental
    Arm Description
    Subjects randomized to 60 units/kg
    Intervention Type
    Drug
    Intervention Name(s)
    Taliglucerase alfa
    Intervention Description
    Taliglucerase infusion every two weeks for 21 months
    Primary Outcome Measure Information:
    Title
    Spleen Volume
    Description
    Spleen volume measured by MRI
    Time Frame
    60 months
    Secondary Outcome Measure Information:
    Title
    Liver Volume
    Description
    Liver volume by MRI
    Time Frame
    60 months
    Title
    Platelet Count
    Description
    Platelet count measure annually
    Time Frame
    60 months
    Title
    Hemoglobin
    Description
    Hemoglobin measure yearly
    Time Frame
    60 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility 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.

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    27499018
    Citation
    Zimran A, Duran G, Giraldo P, Rosenbaum H, Giona F, Petakov M, Terreros Munoz E, Solorio-Meza SE, Cooper PA, Varughese S, Alon S, Chertkoff R. Long-term efficacy and safety results of taliglucerase alfa through 5years in adult treatment-naive patients with Gaucher disease. Blood Cells Mol Dis. 2019 Sep;78:14-21. doi: 10.1016/j.bcmd.2016.07.002. Epub 2016 Jul 18.
    Results Reference
    derived

    Learn more about this trial

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

    We'll reach out to this number within 24 hrs