A Study of Obeticholic Acid Compared to Placebo in children with Biliary Atresia
Research type
Research Study
Full title
A Randomized, Double-blind, Placebo-controlled, Phase 2/3 Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Obeticholic Acid Compared to Placebo in Pediatric Subjects with Biliary Atresia, Post-hepatoportoenterostomy
IRAS ID
1009427
Contact name
Scott Birnbaum
Contact email
Sponsor organisation
Intercept Pharmaceuticals, Inc.
Eudract number
2023-503926-37
Clinicaltrials.gov Identifier
Research summary
The Sponsor of this study, Intercept Pharmaceuticals, Inc. has already developed an investigational drug, obeticholic acid (also known as Ocaliva® or OCA) for the treatment of liver diseases. Intercept plans to study OCA and its effect on biliary atresia (BA) in children and teenagers who have had the Kasai procedure. OCA is a man-made version of a natural substance, bile acid, which is made in the liver. Bile acids are used by the body to help with digestion and have additional effects on the liver function. The purpose of this study is to find out how safe and effective OCA may be in treating children and young adults with BA who have had the Kasai procedure.
Approximately 144 children and teenagers up to 18 years of age will be involved in this study globally. About 50 clinics will take part in the study, including in the United Kingdom. Study participation will last up to approximately 17 months. Participants will be randomised to receive either 0.1-mg mini-tablet or matching placebo (dummy tablet containing no OCA) and 1.5-mg tablet or matching placebo tablet. Starting on Day 1 and over the next 6 weeks, participants may receive up to 3 different doses of OCA or placebo, increasing over time as instructed by the site staff. Eligibility for each dose increase will be determined based on blood test results and other safety results. The OCA or placebo will be taken by mouth on a daily basis for up to 17 months. A patient diary will be provided to record the number of tablets taken each day. Participants will undergo a number of investigations during their participation including physical examination, blood and urine testing, ECG, and ultrasound imaging of the liver (though this may not be conducted at all sites).
REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
24/LO/0272
Date of REC Opinion
25 Jul 2024
REC opinion
Further Information Favourable Opinion