N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy

• Register: ClinicalTrials.gov
• Last refreshed on: 1 April 2024
• Main ID: NCT03499249
• Date of registration: 04/04/2018
• Prospective Registration: Yes
• Primary sponsor: Baylor College of Medicine
• Public title: N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy
• Scientific title: A Phase 2 Trial of N-Acetylcysteine in Biliary Atresia After Kasai Portoenterostomy
• Date of first enrolment: May 18, 2018
• Target sample size: 13
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03499249
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Sanjiv Harpavat, MD. PhD
• Affiliation: Baylor College of Medicine

Key inclusion & exclusion criteria

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



Age minimum: 0 Days
Age maximum: 90 Days
Gender: All

Health Condition(s) or Problem(s) studied

Biliary Atresia

Intervention(s)

Drug: N-Acetyl cysteine

Primary Outcome(s)

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]

Secondary Outcome(s)

Number of Patients Experiencing Sentinel Events in the First 2 Years of Life [Time Frame: First two years of life]

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]

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)]

Secondary ID(s)

135796

H-40962

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 10/01/2024
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03499249