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.

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

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2003-04-30

Study Completion Date

2006-08-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Pompe Disease Late-Onset Glycogen Storage Disease Type II GSD 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

30 mg/kg qow f and 40 mg/kg qow

Interventions

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Myozyme

30 mg/kg qow f and 40 mg/kg qow

Intervention Type BIOLOGICAL

Other Intervention Names

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Alglucosidase alfa

Eligibility Criteria

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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.

Minimum Eligible Age

16 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No