A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
- Registration Number
- NCT01422187
- Lead Sponsor
- Pfizer
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 19
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Taliglucerase alfa 30 units/kg Taliglucerase alfa Subjects randomized to receive 30 units/kg Taliglucerase alfa 60 units/kg Taliglucerase alfa Subjects randomized to 60 units/kg
- Primary Outcome Measures
Name Time Method Spleen Volume 60 months Spleen volume measured by MRI
- Secondary Outcome Measures
Name Time Method Hemoglobin 60 months Hemoglobin measure yearly
Liver Volume 60 months Liver volume by MRI
Platelet Count 60 months Platelet count measure annually