Read the Latest
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 international multi-center study evaluates the drug KB195 when used to treat urea cycle disorders (UCDs). Rare genetic conditions, UCDs cause accumulation of high levels of ammonia in the blood, which can lead to brain damage and other serious problems. Researchers will be monitoring to see if KB195 can reduce the amount of ammonia in blood of people with UCD.
Subject to certain exclusion criteria, candidates must have a confirmed UCD diagnosis for which the standard of care (dietary control) has not controlled the disease well.
Males and Females: Ages 12 to 70
All participants in the study will receive KB195, which will be provided as a powder to be mixed with water before being taken orally, twice a day. The dosage level will increase on days 5, 9 and 13. The final dose level will continue until the end of the dosing period (day 56). For screening, physical examinations, blood testing, and other evaluations, 9 study visits are scheduled. Additionally researchers will contact participants by telephone at specific intervals.
Visits: At least 10
Duration: About 4 months
Study Description at National Institutes of Health
Urea Cycle Disorders
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Mark Tumblin, Clinical Research Coordinator
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.