Tolerability of Enteral NAC in Infants (NCT06260566) | Clinical Trial Compass
Not Yet RecruitingPhase 1
Tolerability of Enteral NAC in Infants
12 participantsStarted 2026-10
Plain-language summary
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.
Who can participate
Age range
122 Days – 273 Days
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
No emesis within 30 minutes of administration of at least 3 of 4 total doses of oral NAC