Children's Hospital is part of the UPMC family.
Be safe anytime, anywhere.
To find a pediatrician or pediatric specialist, please call 412-692-7337 or search our directory.
A resource for our network of referring physicians.
For more information about research, please call our main office at 412-692-6438.
Children's Hospital is ranked One of America's Best Children's Hospitals.
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:
Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
With myCHP, you can request appointments, review test results, and more.
For questions about a hospital bill call:
To pay your bill online, please visit UPMC's online bill payment system.
Interested in giving to Children's Hospital? Visit Children's Hospital of Pittsburgh Foundation's website to make a donation online.