Children's Hospital is part of the UPMC family.
Through this study researchers will evaluate the drug PRX-102 as an enzyme-replacement therapy for Fabry disease and particularly its ability to decrease the negative effects Fabry disease has on kidney function.
Subject to certain exclusion criteria, the study is accepting patients of both genders, ages 18 to 60, who have a confirmed diagnosis of Fabry disease and who are currently being treated with an enzyme, agalsidase beta, also known as Fabrazyme, for reduced kidney function believed to be associated with the disease.
Males and Females: Ages 18 to 60
In this study, participants will be assigned to either continue their current therapy or be given PRX-102. The determination will be made by random selection, although twice as many enrollees will receive PRX-102. Participants will visit the clinic every 2 weeks over a 2-year period for a total of 53 intravenous infusions of either PRX-102 or Fabrazyme. During some visits, physical examinations, test procedures, and imaging scans will be performed.
Duration: 2 years
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Michele Graham, RN
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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