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

A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00763932

Enrollment

Registered

2008-10-01

Start date

2003-04-30

Completion date

2006-07-31

Last updated

2014-02-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Glycogen Storage Disease Type II, Pompe Disease Infantile-Onset

Brief 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

Interventions

BIOLOGICALMyozyme

10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

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

Design outcomes

Primary

Measure	Time frame
The objective of this extension study was to monitor the long-term safety and efficacy	3 years

Countries

France, South Africa, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026