Back to resultsExtension 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.
Primary Purpose
Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Myozyme
Sponsored by
About this trial
This is an interventional treatment trial for Pompe Disease Late-Onset
Eligibility Criteria
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.
Sites / Locations
- University of Texas Health Science Center at San Antonio
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
1
Arm Description
Outcomes
Primary Outcome Measures
The objective of this extension study was to monitor the long-term safety and efficacy of a single patient
Secondary Outcome Measures
Full Information
NCT ID
NCT00765414
First Posted
October 2, 2008
Last Updated
February 4, 2014
Sponsor
Genzyme, a Sanofi Company
1. Study Identification
Unique Protocol Identification Number
NCT00765414
Brief Title
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.
Official Title
An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies
Study Type
Interventional
2. Study Status
Record Verification Date
February 2014
Overall Recruitment Status
Completed
Study Start Date
April 2003 (undefined)
Primary Completion Date
April 2006 (Actual)
Study Completion Date
August 2006 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
Genzyme, a Sanofi Company
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Myozyme
Other Intervention Name(s)
Alglucosidase alfa
Intervention Description
30 mg/kg qow f and 40 mg/kg qow
Primary Outcome Measure Information:
Title
The objective of this extension study was to monitor the long-term safety and efficacy of a single patient
Time Frame
3 years
10. Eligibility
Sex
Female
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
Facility Name
University of Texas Health Science Center at San Antonio
City
San Antonio
State/Province
Texas
Country
United States
12. IPD Sharing Statement
Learn more about this trial
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.
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