COVID-19 Information Read the Latest
Children's Hospital is part of the UPMC family.
Our Sites
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 research study, being carried out at Children’s Hospital of Pittsburgh of UPMC and Presbyterian Hospital of UPMC, is designed to determine whether carbamazepine (CBZ), a drug which has been used safely for many years for seizures and depression, can reduce the severity of liver disease that occurs in some people with the genetic disorder known as alpha-1 antitrypsin (AT) deficiency, or ATD. In this disorder a genetic mutation results in the formation of an abnormal protein that accumulates in liver cells. This accumulation is toxic to the liver cells and therefore leads to gradual scarring (fibrosis) and ultimately failure of the liver to function properly (cirrhosis). Researchers from Children’s Hospital have shown that CBZ stimulates a process that removes abnormal proteins from cells in animal models of ATD. CBZ, also known by the trade name Tegretol-XR, is currently FDA approved for use by children in the treatment of seizures, depression and facial pain due to nerve inflammation.
Subject to certain exclusion criteria, this study is accepting patients of both genders, ages 14 through 80, with liver disease due to alpha-1 antitrypsin deficiency. Males: 14 years through 80 years Females: 14 years through 80 years
Participants will be randomly assigned to groups that will either take CBZ or a placebo, prepared in identical appearing capsules. Two thirds of the subjects will take CBZ and one-third will take placebo. Dosages will be increased weekly for the first five weeks, and monitoring of the drug level in the blood will be done throughout the study to reduce side effects. Liver biopsies and measurement of liver pressure will be done at the beginning of the study and at week 52, after which the study medication will be stopped. Visits: 7 Duration: Approximately 1 year
National Institutes of Health Novartis
Study Description at National Institutes of Health News Release about this Study
For information or to enroll, please contact: Adam Kufen, RN, BS, CCRC 1-855-428-2281 liverstudy@chp.edu
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.