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, a Sanofi Company1 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Pompe Disease Late-Onset Glycogen Storage Disease Type II GSD II

Interventions

MyozymeBIOLOGICAL

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

Eligibility

Sex: FEMALEMin age: 16 Years

Medical Language ↔ Plain English

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.

Locations (1)

University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

Outcomes

Primary Outcomes

The objective of this extension study was to monitor the long-term safety and efficacy of a single patient

Time frame: 3 years

Data from ClinicalTrials.gov

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