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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 February 2015
Main ID:  NCT00763932
Date of registration: 30/09/2008
Prospective Registration: No
Primary sponsor: Genzyme, a Sanofi Company
Public title: 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
Scientific title: A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies
Date of first enrolment: April 2003
Target sample size: 7
Recruitment status: Completed
URL:  http://clinicaltrials.gov/show/NCT00763932
Study type:  Interventional
Study design:  Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment  
Phase:  Phase 2
Countries of recruitment
France South Africa United States
Contacts
Name:     Medical Monitor
Address: 
Telephone:
Email:
Affiliation:  Genzyme, a Sanofi Company
Key inclusion & exclusion criteria

Inclusion Criteria:

- 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 Criteria:

- 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



Age minimum: 18 Years
Age maximum: N/A
Gender: Both
Health Condition(s) or Problem(s) studied
Glycogen Storage Disease Type II
Pompe Disease Infantile-Onset
Intervention(s)
Biological: Myozyme
Primary Outcome(s)
The objective of this extension study was to monitor the long-term safety and efficacy [Time Frame: 3 years]
Secondary Outcome(s)
Secondary ID(s)
AGLU02003
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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