Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients With Fabry Disease

Fecha de inicio: 1 de febrero de 2001

Inclusion Criteria: * Patients must provide written informed consent * Patients must be at least 16 years old * Patients must have a current diagnosis of Fabry disease and have a clinical presentation consistent of Fabry disease (decreased sweating, Fabry pain, angiokeratoma, etc.) * Patients may not have received enzyme replacement therapy as a treatment for Fabry disease * Patients must have a documented plasma a-galactosidase A (aGAL) activity of \< 1.5 nmol/hr/mL or a documented leukocyte aGAL activity of \< 4 nmol/hr/mg * Patients must have one or more of the following: a serum creatinine measurement of 1.2 to 3 mg/dL (106.1 to 265 umol/L) OR estimated creatinine clearance \< 80 mL/min only if the patient's serum creatinine measurement is \< 1.2 mg/dL * Female patients of childbearing potential must have a negative pregnancy test prior to each dosing and all female patients must use a medically accepted form of contraception Exclusion Criteria: * Patient has undergone or is currently scheduled for kidney transplantation or is currently on dialysis * Patient has acute renal failure * Patient has participated in a study employing an investigational drug within 30 days of study entry * Patient has diabetes mellitus or presence of confounding renal disease * Patient has a history of transient ischemic attack (TIA) or ischemic stroke within 3 months of study entry documented by mild-to-moderate neurological deficit * Patient has critical coronary disease * Patient has congestive heart failure * Patient has severe residual neurological deficit that will confound the detection of new events as determined by an attending neurologist and/or Principal Investigator * Patient is unwilling to comply with the requirements of the protocol or the patient has a medical condition, serious intercurrent illness, or extenuating circumstances that would significantly decrease study compliance, including prescribed follow-up