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

Genzyme

Status and phase: Completed; Phase 2

Conditions: Glycogen Storage Disease Type II; Pompe Disease Infantile-Onset

Treatments: Biological: Myozyme

Study type: Interventional

Funder types: Industry

Identifiers

NCT00763932

AGLU02003

Details and patient eligibility

About

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

Enrollment: 7 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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7 participants in 1 patient group

1

Experimental group

Treatment:

Biological: Myozyme