Tolerability of Enteral NAC in Infants
Biliary atresia (BA) is a neonatal liver disease characterized by impaired bile flow and is the most common indication for pediatric liver transplantation. BA can be treated with the Kasai portoenterostomy (KP), a procedure that attempts to restore bile flow and slow disease progression. However, success of the KP procedure is quite variable, and lack of adjuvant medical therapies following KP is a major gap in pediatric hepatology.

This study begins to explore oral N-acetylcysteine (NAC) as a potential medical therapy in BA by determining whether an oral formulation can be given to infants. The primary objective is to determine tolerability of the oral NAC formulation. The primary outcome is tolerating at least 3 out of 4 total doses without emesis. The Bayesian Optimal Interval Design (BOIN) trial design will be used to determine the maximum tolerated dose of oral NAC. Our secondary objective is to assess palatability of the oral NAC formulation by comparing facial expressions when taking oral NAC versus other medications commonly given to cholestatic infants.
- EligibilityCriteria: Inclusion Criteria:  122-273 days of life at time of enrollment Confirmed diagnosis of biliary atresia based on intraoperative cholangiogram Able to tolerate oral nutrition and medications and not on continuous tube feeds Anticipated inpatient admission of at least 4 days Legal guardian(s) consent to study enrollment after understanding the risks and investigational nature of the study  Exclusion Criteria:  Gestational age of <32 weeks at birth Inability or contraindication to taking oral nutrition Neonatal intensive care unit admission Short bowel, or other malabsorptive, syndrome Decompensated liver disease (INR > 1.3 despite vitamin K administration) Active respiratory infection Severe concurrent illnesses that would interfere with the conduct and/or results of the study Concurrent participation in another drug trial - HealthyVolunteers: No - Gender: All - MinimumAge: 122 Days - MaximumAge: 273 Days - StdAgeList: Child