Studies Open for Enrollment

A Multi-center Study of the Safety and Efficacy of N-Acetylcysteine in the Treatment of Acute Liver Failure Not Caused by Acetaminophen in Pediatric Patients

The purpose of this study is to test whether a drug called N-acetylcysteine (NAC), given as an intravenous infusion, improves liver function in children diagnosed with pediatric acute liver failure (PALF) that is not a result of acetaminophen toxicity. The PALF study will attempt to determine whether NAC may improve liver function when liver failure is caused by other harmful agents, such as infections, other drugs or genetic conditions, or when a specific cause is not identified. Children’s Hospital of Pittsburgh of UPMC is the clinical coordinating center of this study, comprised of a network of 19 liver centers, which includes three international centers.

Candidates
With certain restrictions, boys and girls under the age of 18 diagnosed with acute liver failure, not due to acetaminophen overdoses, may be eligible to participate.
Boys: Newborn through 17 years
Girls: Newborn through 17 years

Requirements
Participants in the study will already be hospitalized and must be enrolled in the PALF Study Group Registry. Participants will be randomly assigned to receive the study drug (N-acetylcysteine) or placebo over 7 days as a continuous intravenous infusion. Data on participants will be collected as part of the PALF Registry. Additional data forms will be collected at 6 months and 12 months following enrollment.
Visits: None beyond patient’s hospital stay
Duration: 1 year

Status: Open for Enrollment

How to Participate
To get started, please contact:
Beverly Bernard, CRNP
412-692-5811

Additional Resources
Study Description at National Institutes of Health
The Pediatric Acute Liver Failure (PALF) Study Web Site

Principal Investigator
Robert H. Squires, Jr., MD
Pediatric Gastroenterology, Hepatology and Nutrition

Source of Support
National Institute of Diabetes and Digestive and Kidney Diseases