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.

Completed · Phase 2

Conditions studied: Pompe Disease Late-Onset, Glycogen Storage Disease Type II GSD II

In brief

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.

Key facts

Study ID

NCT00765414

Run by

Genzyme, a Sanofi Company

People needed

1

Starts

2003-04-01

Expected to finish

2006-08-01

Last updated by the study team

2014-02-05

Who can join

Age: 16 and older. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States

Full record on ClinicalTrials.gov

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