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.

Genzyme

Status and phase

Completed

Phase 2

Conditions

Pompe Disease Late-Onset

Glycogen Storage Disease Type II GSD II

Treatments

Biological: Myozyme

Study type

Interventional

Funder types

Industry

Identifiers

NCT00765414

AGLU02103

Details and patient eligibility

About

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.

Enrollment

1 patient

Sex

Female

Ages

16+ years old

Volunteers

No Healthy Volunteers

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.