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