Study to Assess Efficacy, Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Obeticholic Acid (OCA) Compared to Placebo in Pediatric Participants With Biliary Atresia, Post-hepatoportoenterostomy

NCT ID: NCT06121375

Last Updated: 2025-10-31

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 28 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-09-02
• Study Completion Date: 2025-10-21

Brief Summary

This study will evaluate the efficacy, safety and tolerability, as well as PK/PD of OCA in eligible pediatric participants with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterostomy). The double-blind period comprises of 2 phases: dose titration phase and age expansion treatment phase.

Conditions

Biliary Atresia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Participants receiving OCA

Participants will be randomized to receive OCA (starting at 1.5 milligrams \[mg\] adult equivalent dose \[AED\]) orally, with water, once daily. Dose will be titrated every 2 weeks in a stepwise manner for the first 6 weeks, starting at 1.5 mg AED and titrating through 3 mg AED to a maximum of 5 mg AED, as tolerated; a discussion with the Medical Monitor is encouraged when determining uptitration if considerable signs or symptoms have arisen. Following the 6-week dose titration phase, participants will continue at the tolerated dose for approximately 24 months in Age Expansion Treatment Phase.

Group Type: ACTIVE_COMPARATOR

OCA

Intervention Type: DRUG

OCA will be administered.

Participants receiving Matching placebo

Participants will be randomized to receive matching placebo orally, with water, once daily. Dose will be titrated every 2 weeks in a stepwise manner for the first 6 weeks, starting at 1.5 mg AED and titrating through 3 mg AED to a maximum of 5 mg AED, as tolerated; a discussion with the Medical Monitor is encouraged when determining uptitration if considerable signs or symptoms have arisen. Following the 6-week dose titration phase, participants will continue at the tolerated dose for approximately 24 months in Age Expansion Treatment Phase.

Group Type: PLACEBO_COMPARATOR

Matching Placebo

Intervention Type: DRUG

Matching Placebo will be administered.

Interventions

OCA

OCA will be administered.

Intervention Type: DRUG

Matching Placebo

Matching Placebo will be administered.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Male or female pediatric participants from birth to <18 years old. Note: Participants aged <2 years old will not be enrolled until after review of safety data during the planned interim analysis and agreement from the Data Safety Monitoring Board (DSMB) that there is sufficient safety data to enroll this age group.
• Diagnosis of non-syndromic biliary atresia.
• Demonstrated successful HPE as defined by total bilirubin <2 milligrams per deciliter (mg/dL) (34.2 micromoles per liter [μmol/L]) at least 3 months post-HPE procedure.

Exclusion Criteria

• Prior liver transplant or active status on transplant list.
• Participants diagnosed with biliary atresia splenic malformation (BASM).
• Conjugated (direct) bilirubin ≥ upper limit of normal (ULN) of site-specific reference range. If conjugated bilirubin is not available: total bilirubin ≥2 mg/dL (34.2 mol/L).
• Platelets <120,000/μL
• International normalized ratio (INR) ≥1.5.
• Current or history of complications of decompensated chronic liver disease including:

1. Gastroesophageal varices and/or variceal bleeding

2. Clinically evident ascites related to portal hypertension

3. Hepatic encephalopathy

4. Prior placement of portosystemic shunt

5. Hepatopulmonary syndrome or portopulmonary hypertension

6. Hepatorenal syndrome

7. Any evidence of portal hypertension based on imaging (e.g., cavernous transformation of portal vein, abdominal varices, etc.)

8. Hepatocellular carcinoma

9. Childs-Pugh B or C

• Height and weight Z-score <-2 per site-specific reference ranges.
• Acholic (pale) stools.
• Aspartate aminotransferase (AST) >4x ULN.
• Alanine aminotransferase >4x ULN
• GGT >500 Units per Liter (U/L)
• On anticoagulation therapy
• Albumin <3.5 grams per deciliter (g/dL).
• Inability to swallow tablets (i.e., tablet or mini-tablet formulations).

• Minimum Eligible Age: 1 Day
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Intercept Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Lynda Szczech, MD

Role: STUDY_DIRECTOR

Intercept Pharmaceuticals

Locations

Queensland Childrens Hospital

South Brisbane, Queensland, Australia

Women's and Children's Hospital

North Adelaide, South Australia, Australia

Royal Childrens Hospital

Parkville, Victoria, Australia

Alberta Childrens Hospital

Calgary, Alberta, Canada

Stollery Children's Hospital

Edmonton, Alberta, Canada

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

Guangzhou Women And Childrens Medical Center

Guangzhou, , China

Children's Hospital of Fudan University

Shanghai, , China

Childrens Hospital of Shanghai

Shanghai, , China

Children's Hospital of Shanxi

Taiyuan, , China

Queen Mary Hospital

Hong Kong, , Hong Kong

Hadassah Medical Center

Jerusalem, , Israel

Shaare-Zedek Medical Center

Jerusalem, , Israel

Hospital Raja Perempuan Azinab II

Kota Bharu, Kelantan, Malaysia

University Malaya Medical Center

Kuala Lumpur, , Malaysia

Starship Child Health

Auckland, , New Zealand

KK Women's and Children's Hospital

Singapore, , Singapore

Taichung Veterans General Hospital

Taichung, , Taiwan

National Chen Kung University Hospital

Tainan City, , Taiwan

National Taiwan University Hospital

Taipei, , Taiwan

Linkou Chang Gung Memorial Hospital

Taoyuan District, , Taiwan

Akdeniz Üniversitesi Tıp Fakültesi Hastanesi Pediatrik Gastroenteroloji

Konyaalti, Antalya, Turkey (Türkiye)

Ege Üniversitesi Hastanesi Pediatrik Gastroenteroloji Bölümü

Bornova, İzmir, Turkey (Türkiye)

Hacettepe Universitesi ihsan Dogramaci Cocuk Hastansesi

Ankara, , Turkey (Türkiye)

Countries

Australia; Canada; China; Hong Kong; Israel; Malaysia; New Zealand; Singapore; Taiwan; Turkey (Türkiye)

Other Identifiers

747-308

Identifier Type: -

Identifier Source: org_study_id