Trial · Interventional

CompletedPhase 2Results postedRegistry data

N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy

NCTNCT03499249Other idH-40962Other id135796

Open at ClinicalTrials.govAsk about this trial

This trial does not test a peptide that PepBank tracks.

Names Glutathione, does not test it. It appears in the summary.

Overview

Status: Completed
Phase: Phase 2
Enrollment: 13 participants (actual)
Sponsor: Baylor College of Medicine · Other
Design: Allocation not applicable · Open label (no masking)
Countries: 1 country
United States
Start: 18 May 2018
Primary completion: 31 Oct 2022
Completion: 23 Mar 2024
First posted: 17 Apr 2018
Last update posted: 26 Mar 2024
Conditions: Biliary Atresia

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

2018202020222024

Ph 2
N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy
NCT03499249
18 May 2018 to 23 Mar 2024

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

Biliary atresia (BA) is a devastating liver disease of infancy, characterized by bile duct obstruction leading to liver fibrosis, cirrhosis, and eventual need for transplantation in most cases. BA is treated with Kasai portoenterostomy (KP). KPs can achieve bile drainage and improve outcomes. However, even with standard evidence of "good bile flow," bile flow rarely normalizes completely and liver disease continues to progress. In this study, the investigators test whether intravenous N-acetylcysteine (NAC) can improve bile flow after KP. The rationale is that NAC leads to synthesis of glutathione, which is a powerful stimulator of bile flow. The primary objective is to determine whether NAC normalizes total serum bile acid (TSBA) concentrations within 24 weeks of KP. Achieving normal TSBAs is uncommon with current standard-of-care, and is predicted to be associated with better long-term outcomes. The secondary objectives are to describe how other parameters commonly followed in BA change with NAC therapy, as well as report adverse events occurring with therapy and in the first two years of life. This study follows the "minimax" Phase 2 clinical trial design.

Interventions

N-Acetyl cysteineDrug

Arms1

Experimental
N-Acetylcysteine Treatment
Will receive continuous intravenous NAC therapy (6.25 mg/kg/hour of 10 mg/ml solution, or 0.625 ml/kg/hour, to give 150 mg/kg/day), starting within 24 hours of completion of KP and lasting for a total of 7 days

Primary outcomes1

Outcomes the sponsor listed as primary.

Number of Patients With Biliary Atresia (BA) Achieving Total Serum Bile Acids Less Than or Equal to 10 *U*Mol/L Within 24 Weeks of Kasai Portoenterostomy (KP)
Time frame · Within 24 weeks after KP
Registry description
Expected is \~5% of participants based on historical controls (see protocol for summary of historical controls); a higher number is a better outcome

Secondary outcomes(3)

Aspartate Aminotransferase (AST), Alanine Aminotransferase (ALT), Gamma-glutamyltransferase (GGT) Fold, and Conjugated Bilirubin (Bc) Change Above Baseline at 3 Days and 7 Days After KP (During Treatment)
Time frame · 3 days after KP compared to baseline (before KP); 7 days after KP compared to baseline (before KP)
Registry description
fold-change from baseline (ratio); a fold-change \<1 is a better outcome.
Number of Patients Experiencing Sentinel Events in the First 2 Years of Life
Time frame · First two years of life
Registry description
Events include Nasogastric (NG) feeds or Total Parental Nutrition (TPN) initiation, Splenomegaly (based on ultrasound findings), Thrombocytopenia (platelets \<150,000/mm3), Ascites (recorded when diuretics were needed for fluid accumulation), GI bleed (varices documented by endoscopy), Portal hypertension (one of the following: ascites, or combination of splenomegaly and thrombocytopenia), and Liver transplant or death; units are number of patients, higher numbers are worse outcomes.
Number of Patients With Adverse Events Possibly Related to NAC, Including Rash, Urticaria, Pruritus, Tachycardia, Hypotension, Vomiting, Edema, Anaphylaxis, and Intravenous Line Issues
Time frame · Within four weeks after KP
Registry description
Units are number of patients, higher number is worse outcome

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· Within 24 weeks after KP

Number of Patients With Biliary Atresia (BA) Achieving Total Serum Bile Acids Less Than or Equal to 10 UMol/L Within 24 Weeks of Kasai Portoenterostomy (KP)

Group	Value (Participants)
N-Acetylcysteine Treatment	0

Secondary and other outcome results(3)

Secondary outcomeMean· 3 days after KP compared to baseline (before KP); 7 days after KP compared to baseline (before KP)

Aspartate Aminotransferase (AST), Alanine Aminotransferase (ALT), Gamma-glutamyltransferase (GGT) Fold, and Conjugated Bilirubin (Bc) Change Above Baseline at 3 Days and 7 Days After KP (During Treatment)

Group	Value (fold change over baseline)	Spread
N-Acetylcysteine Treatment	1.5	0.5

Secondary outcomeCount of participants· First two years of life

Number of Patients Experiencing Sentinel Events in the First 2 Years of Life

Group	Value (Participants)
N-Acetylcysteine Treatment	4

Secondary outcomeCount of participants· Within four weeks after KP

Number of Patients With Adverse Events Possibly Related to NAC, Including Rash, Urticaria, Pruritus, Tachycardia, Hypotension, Vomiting, Edema, Anaphylaxis, and Intravenous Line Issues

Group	Value (Participants)
N-Acetylcysteine Treatment	1

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
N-Acetylcysteine TreatmentWill receive continuous intravenous NAC therapy (6.25 mg/kg/hour of 10 mg/ml solution, or 0.625 ml/kg/hour, to give 150 mg/kg/day), starting within 24 hours of completion of KP and lasting for a total of 7 days N-Acetyl cysteine: Intravenous NAC therapy (6.25 mg/kg/hour of 10 mg/ml solution, or 0.625 ml/kg/hour, to give 150 mg/kg/day), starting within 24 hours of completion of KP and lasting for a total of 7 days	0 / 13	5 / 13	11 / 13

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

DerivedHarpavat S, Borovsky KA, Scheurer ME, Cavallo L, Erhiawarie FE, Vasudevan S, Vogel AM, Cerminara D, Tessier EM, Patel KR, Devaraj S, Shneider BL. A phase 2 trial of short-term intravenous N-acetylcysteine in biliary atresia after Kasai portoenterostomy. Hepatol Commun. 2025 Jun 9;9(7):e0729. doi: 10.1097/HC9.0000000000000729. eCollection 2025 Jul 1. PMID 40489761

Eligibility

Who could join, as posted.

Sex: All
Age: from 0 Days up to 90 Days
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

Inclusion Criteria: 1. Age less than or equal to 90 days at time of KP (standard age range in which KPs are performed) 2. BA diagnosis made by intraoperative cholangiography and KP performed at Texas Children's Hospital, Texas Medical Center Campus 3. Legal guardian(s) sign consent after understanding risks and investigational nature of study Exclusion Criteria: 1. Decompensated liver disease (INR \>1.3) despite parenteral Vitamin K administration) 2. KP not performed for any reason (i.e., normal intraoperative cholangiography, or liver found to be too diseased intraoperatively to proceed with KP) 3. Active respiratory infection 4. Renal impairment, as defined by having an eGFR \< 60 mL/min/1.73m2 or creatinine clearance \< 60 mL/min (https://www.niddk.nih.gov/health-information/communication-programs/nkdep/laboratory-evaluation/glomerular-filtration-rate-calculators/children-conventional-units) 5. Presence of severe concurrent illnesses, such as pulmonary (i.e., bronchopulmonary dysplasia), neurological, cardiovascular, metabolic, endocrine, and renal disorders, which may be congenital or acquired, that would interfere with the conduct and results of the study

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