COVID-19: Safety, Testing, News Alerts, and More.
Read the Latest Update
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.
Ranked #9 Nationally by U.S. News & World Report.
This multi-center research program evaluates the effectiveness, potential side effects, and how the body processes the drug maralixibat when used as a therapy for progressive familial intrahepatic cholestasis (PFIC) in children, adolescents and teens over an extended period of time. PFIC is a rare genetic condition in which cells in the liver cannot release bile, a fluid that helps the body digest food. As a result, bile builds up in the liver, leading to symptoms such as jaundice, itching, impaired growth, and delayed puberty.
This study (MRX-503) is open to patients who have PFIC and who participated in an earlier maralixibat study protocol (MRX-502).
Males and Females: 1 to 18 years of age
The study drug will be given as an oral solution taken twice a day. Each day, participants or their caregivers will be required to complete study questionnaires using an electronic data logging device provided by the study team. Participants will receive examinations, including blood and urine sampling, and ultrasound imaging at specific intervals.
Duration: 2 years
Study Description at the National Institutes of Health
Progressive Familial Intrahepatic Cholestasis
Robert Squires, MD
For more information about the study or enrollment, please contact:
Clinical Research Nurse Coordinator Amanda Blasko, RN, BSN
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
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? Support the hospital by making a donation online, joining our Heroes in Healing monthly donor program, or visiting our site to learn about the other ways you can give back.