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Tolerability of Enteral NAC in Infants

NCT06260566 · Status: NOT_YET_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-03-04

No results posted yet for this study

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.

Conditions

  • Biliary Atresia

Interventions

DRUG

N-Acetylcysteine

This is a phase 1 study of tolerance of a novel delivery method of oral NAC medication in infants with biliary atresia. The study consists of two parts: * Administering applesauce alone (lead-in): In study days 1-2, infants will receive one infant spoon (approximately ½ teaspoon) of plain applesauce by mouth twice a day. If the infant tolerates 3-4 of 4 total doses without emesis, the infant will progress to the final two days of the study. * Administering applesauce plus NAC (for infants who tolerate applesauce in study days 1-2): In study days 3-4, infants will receive an individualized dose of powdered oral NAC mixed into one infant spoon of applesauce by mouth twice a day for a maximum of four doses of NAC medication. 4 doses will be trialed: 150 mg/kg/day, 240 mg/kg/day, 330 mg/kg/day, and 420 mg/kg/day using BOIN study design for dose escalation.

Sponsors & Collaborators

  • Baylor College of Medicine

    collaborator OTHER

  • Sanjiv Harpavat

    lead OTHER

Principal Investigators

  • Sanjiv Harpavat, M.D., PH.D. · Baylor College of Medicine - Texas Children's Hospital

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
122 Days
Max Age
273 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-10-31
Primary Completion
2030-10-31
Completion
2031-10-31
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06260566 on ClinicalTrials.gov