Status:
COMPLETED
Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Pompe Disease Infantile-Onset
Glycogen Storage Disease Type II
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac c...
Eligibility Criteria
Inclusion
- The patient was enrolled in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502
- The patient's legal guardian(s) provided written informed consent prior to any study related procedures being performed
- The patient and his/her guardian(s) were able to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.
Exclusion
- Patients were excluded from this study if they did not meet the specific inclusion criteria, or if the patient experienced any unmanageable AE in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation), due to Synpac rhGAA therapy, that would preclude continuing therapy
Key Trial Info
Start Date :
April 1 2003
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2006
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT00763932
Start Date
April 1 2003
End Date
July 1 2006
Last Update
February 5 2014
Active Locations (8)
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1
Children's Hospital of Oakland
Oakland, California, United States
2
Children's Hospital of Orange Country
Orange, California, United States
3
University of Nebraska Medical Center
Omaha, Nebraska, United States
4
Institute for Genetic Medicine, Saint Peter's University Hospital
New Brunswick, New Jersey, United States