COVID-19: Vaccines, Testing, and Treatment
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 compares the effects of glycerol phenylbutyrate and sodium phenylbutyrate to manage 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. Glycerol phenylbutyrate, known by the trade name Ravicti® and sodium phenylbutyrate, known as Buphenyl®, work similarly by helping to remove unwanted ammonia from the body. Both Raviciti, a liquid, and Buphenyl, a tablet or powder, can be taken orally or by feeding tube.
Candidates must have a suspected or confirmed UCD diagnosis without having been treated over a long period of time with either of the study drugs.
Males and Females: All ages
By random selection, participants will be assigned to a treatment arm that starts with either Raviciti or Buphenyl to be taken 3 times a day for 4 weeks. After that, participants who started on Buphenyl will transition to Raviciti for 8 more weeks and those who started on Raviciti will continue on the drug. During the dosing periods, participants will return to the clinic for examinations, blood tests and other evaluations. A 12-week safety extension period will provide additional examinations and monitoring.
Duration: About 25 weeks
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:
Jessica Lindenberger, MSN, CRNP, FNP-C
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.