At a glance
ClinicalIndex Comparison Record- ✓Age at least 16 years
- ✓Confirmed diagnosis of Fabry disease with clinical presentation (decreased sweating, Fabry pain, angiokeratoma, etc.)
- ✓No prior enzyme replacement therapy for Fabry disease
- ✓Documented alpha-galactosidase A deficiency: plasma activity <1.5 nmol/hr/mL OR leukocyte activity <4 nmol/hr/mg
- ✕Current dialysis or scheduled kidney transplantation
- ✕Acute renal failure
- ✕Diabetes mellitus or confounding renal disease
- ✕Recent TIA or ischemic stroke (within 3 months) with mild-to-moderate neurological deficit
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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
In Brief
A Phase 4 clinical trial evaluating Fabrazyme (agalsidase beta) and Placebo for Fabry Disease. Completed, enrolled 82 participants across 26 sites in 6 countries.
Detailed 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.
Study Details
Timeline
Interventions
1mg/kg Fabrazyme (agalsidase beta) every 2 weeks
1 mg/kg placebo intravenously every 2 weeks