Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

A Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal Disease

This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.

Inclusion Criteria:

• provided written informed consent prior to any study-related procedures being performed.
• be ≥16 years old.
• have a current diagnosis of Fabry disease (defined as abnormal α-galactosidase (α GAL) enzyme levels or Fabry genotype).
• have one of the following clinical conditions present at enrollment: serum creatinine level greater than 3.0 mg/dL (an average of two values at least one week apart), or be currently on dialysis, or be status post kidney transplant by greater than 3 months.
• have the ability to comply with the requirements of the protocol
• have a negative pregnancy test, if a female patient of childbearing potential. In addition, all female patients of childbearing potential must use a medically accepted method of contraception throughout the study.

Exclusion Criteria:

• if they did not meet the specific inclusion criteria.
• if they had participated in a study employing an investigational drug within 30 days of the start of their participation in this trial.
• had previously received enzyme replacement therapy (ERT) for their Fabry disease.
• had diabetic nephropathy.
• were pregnant or lactating.
• were unwilling to comply with the requirements of the protocol.