A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
Condition: Fabry Disease
MeSH terms: Fabry Disease
Intervention: Fabrazyme (agalsidase beta); Placebo
Intervention description: 1mg/kg Fabrazyme (agalsidase beta) every 2 weeks; 1 mg/kg placebo intravenously every 2 weeks
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 82.0
Sex: All
Minimum age: 16 Years
Healthy volunteers: No
Keywords: a-Galactosidase A; aGAL; r-haGAL; Fabry; GL-3; Fabrazyme

Summary:

      People with Fabry disease have an alteration in their genetic material (DNA) which causes a
      deficiency of the a-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps
      to breakdown and remove certain types of fatty substances called "glycolipids." These
      glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids
      build up in various tissues such as the liver, kidney, skin, and blood vessels because
      a-galactosidase A is not present, or is present in small quantities. The build up of
      glycolipid ("globotriaosylceramide" or "GL-3") levels in these tissues in particular is
      thought to cause the clinical symptoms that are common to Fabry disease. This study will test
      the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.
    

Eligibility criteria:

        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