Participants are being recruited for a clinical trial to study an investigational enzyme replacement therapy for Fabry disease. The goal of this trial is to determine the efficacy and safety of recombinant human alpha-galactosidase A (Fabrazyme) on the progression of Fabry disease. The study is being conducted worldwide. Patients will be enrolled into one of two treatment arms, and the trial will last for up to 29 months.
Fabry disease is a lysosomal storage disorder in which patients lack the enzyme alpha-Galactosidase. It is X-linked recessive, which means the mother carries the trait for Fabry disease and has a 50 percent chance of passing it on to her children. Fathers pass it on to all of their daughters. Men tend to be more severely affected, but women can develop severe symptoms as well. Some of the more common clinical symptoms include pain and burning sensations in the hands and feet, angiokeratoma (a spotted, dark red skin rash), decreased ability to sweat, kidney failure, cardiac disease and stroke. There is a simple blood test available to determine whether you carry the Fabry gene.
For additional information about the clinical trial, speak to a staff member at Parexel International at (843) 795-8250 or (866) 651-8245.
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