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Tolerability of Enteral NAC in Infants

ConditionBiliary Atresia
Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age122-273
SponsorSanjiv Harpavat

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • N-Acetylcysteine

    Drug

    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.

Treatment groups

12 Participants
are divided into 1 treatment group
Group A: Infants with Biliary AtresiaExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

No emesis within 30 minutes of administration of at least 3 of 4 total doses of oral NAC

No emesis with administration of at least 3 of 4 total doses of oral NAC

Time frame
within 30 minutes

Secondary outcomes

1

Oral NAC palatability compared to clinically indicated medications using the facial expression scale

Facial expression scale uses visual facial expressions scored 0-5 with higher scores indicating worse outcome

Time frame
at the time of adminsitration
2

Oral NAC-specific adverse event evaluation

Adverse event severity grade scale scored 1 - 5 with 5 indicating worse outcome

Time frame
28 days

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Sanjiv Harpavat

Lead sponsor

Baylor College of Medicine

Sponsor institution

Baylor College of Medicine

Collaborator

Source ClinicalTrials.gov

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