Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.
Phase 2
Completed
- Conditions
- Glycogen Storage Disease Type II GSD IIPompe Disease Late-Onset
- Interventions
- Biological: Myozyme
- Registration Number
- NCT00765414
- Lead Sponsor
- Genzyme, a Sanofi Company
- Brief Summary
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Female
- Target Recruitment
- 1
Inclusion Criteria
- Provide written informed consent prior to participating in any study related procedures;
- Currently enrolled in Protocol AGLU01402
- Have the ability to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.
Exclusion Criteria
- Was pregnant or unwilling to use approved birth control during the course of the study;
- Had experienced any unmanageable AEs under Protocol AGLU01402 (as determined and agreed upon by the Principal Investigator and sponsor) due to rhGAA that would preclude continuing ERT;
- Was participating in any other investigational study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description 1 Myozyme -
- Primary Outcome Measures
Name Time Method The objective of this extension study was to monitor the long-term safety and efficacy of a single patient 3 years
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
University of Texas Health Science Center at San Antonio
🇺🇸San Antonio, Texas, United States