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Clinical Trial 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 cell line


Clinical Trial Description

n/a


Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT00763932
Study type Interventional
Source Sanofi
Contact
Status Completed
Phase Phase 2
Start date April 2003
Completion date July 2006

See also
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Active, not recruiting NCT05164055 - Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) Phase 4
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Active, not recruiting NCT02635269 - Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy N/A
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Completed NCT02801539 - Respiratory Muscle Training in L-Onset Pompe Disease (LOPD) N/A