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.

Phase 2

Completed

• Conditions: Glycogen Storage Disease Type II GSD II; Pompe Disease Late-Onset
• Interventions: Biological: Myozyme

• Registration Number: NCT00765414
• Lead Sponsor: Genzyme, a Sanofi Company

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2003-04
• Primary Completion: 2006-04
• Study Completion: 2006-08
• Study First Submitted: 2008-10-02
• Study First Submitted QC: 2008-10-02
• Study First Posted: 2008-10-03
• Status Verified: 2014-02
• Last Update Submitted: 2014-02-04
• Last Update Posted: 2014-02-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Female
• Target Recruitment: 1

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	Myozyme	-

Primary Outcome Measures

Name	Time	Method
The objective of this extension study was to monitor the long-term safety and efficacy of a single patient	3 years

Trial Locations

Locations (1)

University of Texas Health Science Center at San Antonio; 🇺🇸 San Antonio, Texas, United States