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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

7 participants

Study Classification

INTERVENTIONAL

Study Start Date

2003-04-30

Study Completion Date

2006-07-31

Brief Summary

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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

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Detailed Description

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Conditions

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Pompe Disease Infantile-Onset Glycogen Storage Disease Type II

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

Group Type EXPERIMENTAL

Myozyme

Intervention Type BIOLOGICAL

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

Interventions

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Myozyme

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

Intervention Type BIOLOGICAL

Other Intervention Names

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Alglucosidase alfa

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No