Safety and PK/PD Study With Agalsidase Alfa in Canadian Patients With Fabry Disease

• Plasma Gb3 [ Time Frame: 19 weeks ] [ Designated as safety issue: No ]
• Determine pharmacokinetics using standard Pk assessments [ Time Frame: 19 weeks ] [ Designated as safety issue: No ]
  assessed by AUC last/Dose, AUC/dose and Cmax/Dose
• Number of Participants with adverse events as a measure of safety and tolerability [ Time Frame: 19 weeks ] [ Designated as safety issue: Yes ]
  assessed by adverse events, changes in vital signs, physical examinations, concomitant medication usage and anti-agalsidase antibodies

This study is designed to evaluate safety and PK/PD in Canadian Fabry patients.

In 2008, a change in the agalsidase alfa drug substance manufacturing process was made. There are no changes to the drug product formulation, manufacturing site, manufacturing process, or container closure.

This study is designed to provide safety, PD and PK data. The assessment schedule is designed to capture the PK profile of drug uptake in the blood as well the pharmacologic effect which manifests over the course of weeks. Each patient will serve as his own control

Inclusion Criteria:

1. Patient must have a documented diagnosis of Fabry disease.
2. Patient is male and between 18 and 65 years of age.
3. Patient has been treated for Fabry disease for at least 6 months with agalsidase alfa at a dose of 0.2 mg/kg administered by IV EOW.
4. Patient must be willing to remain in the clinic as required by the study and comply with the procedures and evaluations of the study.
5. Patient provides informed consent.

Exclusion Criteria:

1. Patient is unable to be venipunctured and/or tolerate venous access.
2. Patient has tested positive for anti-agalsidase alfa antibodies.
3. Patient had pre-ERT GB3 levels within the normal range (if value is available).
4. Patient is participating in Shire HGT sponsored Study HGT-REP-081. -