Biliary AtresiaPhase 2/3NCT06121375
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
A Phase 2/3 study testing OCA in people with Biliary Atresia, run by Intercept Pharmaceuticals at 24 sites in 10 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 1 day to 18 years
- Conditions the study is for
- Biliary Atresia
- What is being tested?
- OCA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 28 participants
- Where
- 24 sitesin 10 countries
- Study dates
- 2 Sep 2024to 21 Oct 2025
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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:
Show all 23Show fewer
- Gastroesophageal varices and/or variceal bleeding
- Clinically evident ascites related to portal hypertension
- Hepatic encephalopathy
- Prior placement of portosystemic shunt
- Hepatopulmonary syndrome or portopulmonary hypertension
- Hepatorenal syndrome
- Any evidence of portal hypertension based on imaging (e.g., cavernous transformation of portal vein, abdominal varices, etc.)
- Hepatocellular carcinoma
- 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).
Trial locations and contacts
24 sites in 10 countries.
24 sites in 10 countries
China5 sites
Guangzhou Women And Childrens Medical Center
Guangzhou
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310006
Children's Hospital of Fudan University
Shanghai
Childrens Hospital of Shanghai
Shanghai
Children's Hospital of Shanxi
Taiyuan
Taiwan4 sites
Taichung Veterans General Hospital
Taichung
National Chen Kung University Hospital
Tainan
National Taiwan University Hospital
Taipei
Linkou Chang Gung Memorial Hospital
Taoyuan
Australia3 sites
Women's and Children's Hospital
North Adelaide, South Australia, 5006
Royal Childrens Hospital
Parkville, Victoria, 3104
Queensland Childrens Hospital
South Brisbane, Queensland, 4101
Turkey (Türkiye)3 sites
Hacettepe Universitesi ihsan Dogramaci Cocuk Hastansesi
Ankara, 06230
Ege Üniversitesi Hastanesi Pediatrik Gastroenteroloji Bölümü
Bornova, İzmir, 35100
Akdeniz Üniversitesi Tıp Fakültesi Hastanesi Pediatrik Gastroenteroloji
Konyaalti, Antalya, 07050
Canada2 sites
Alberta Childrens Hospital
Calgary, Alberta, T3B 6A8
Stollery Children's Hospital
Edmonton, Alberta
Israel2 sites
Hadassah Medical Center
Jerusalem
Shaare-Zedek Medical Center
Jerusalem
Malaysia2 sites
Hospital Raja Perempuan Azinab II
Kota Bharu, Kelantan, 15586
University Malaya Medical Center
Kuala Lumpur, 59100
About this study
Official 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
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
- Being tested
- OCA, Matching Placebo
- Keywords
- Biliary Atresia, Placebo-controlled, Pharmacokinetics, Pharmacodynamics, Obeticholic Acid, Efficacy, Post-hepatoportoenterostomy
- Medical subject headings
- Biliary Atresia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OCADrug
OCA will be administered.
Matching PlaceboDrug
Matching Placebo will be administered.
| Study group | Role | What they receive |
|---|---|---|
| Participants receiving OCA | Active comparator | 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. Receives: Drug: OCA |
| Participants receiving Matching placebo | Placebo comparator | 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. Receives: Drug: Matching Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Composite Liver-Related Clinical Events
Number of participants experiencing any of the following: All-cause death; Liver transplantation; Hospitalization (≥24 hours) for variceal bleeding, hepatic encephalopathy, or spontaneous bacterial peritonitis; Clinically relevant ascites requiring therapeutic paracentesis have been presented. No clinical outcome events were reported at the time of the study termination; hence It was not possible to conduct the planned primary efficacy analysis.
Measured: Up to Week 48
Change From Baseline in Pediatric End-stage Liver Disease (PELD) Score
The Pediatric End-stage Liver Disease (PELD) score is a scale used to assess the severity of chronic liver disease in pediatric participants younger than 12 years of age. The total PELD score ranges from negative values to positive values, with no absolute minimum or maximum, with higher scores indicating more severe liver disease and greater urgency for liver transplantation. The score is calculated using total bilirubin, international normalized ratio (INR), albumin, age at listing, and growth failure. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was calculated as the last post-baseline value minus the baseline value. A negative change from baseline indicates improvement, while a positive change indicates worsening.
Measured: Baseline and up to Week 48
Change From Baseline in Model of End-stage Liver Disease With Sodium (MELD-NA) Score
The Model of End-stage Liver Disease with Sodium (MELD-Na) score is a scale used to assess the severity of chronic liver disease in participants 12 years of age and older, ranging from 6 (less ill) to 40 (gravely ill). Higher scores indicate more severe liver disease and greater urgency for liver transplantation. The score is derived from total bilirubin, serum creatinine, INR, and serum sodium. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was calculated as the last post-baseline value minus the baseline value. A negative change from baseline indicates improvement, while a positive change indicates worsening.
Measured: Baseline and up to Week 48
Secondary outcomes (8)
Percentage of Participants With Improvement in GGT and Direct Bilirubin (Composite Responder Endpoint)
Participants were considered responders if both of the following criteria were met at EOS: ≥40% reduction from baseline in Gamma Glutamyl Transferase (GGT), and ≥25% reduction from baseline in direct (conjugated) bilirubin. Participants with missing values were considered non-responders.
Measured: Up to Week 48
Change From Baseline in GGT
Blood samples were collected to assess GGT levels. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement.
Measured: Baseline and up to Week 48
Change From Baseline in Total and Direct (Conjugated) Bilirubin
Blood samples were collected to assess direct bilirubin levels. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement.
Measured: Baseline and up to Week 48
Change From Baseline in Endogenous Bile Acids
Plasma samples were collected to assess endogenous bile acids. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement.
Measured: Baseline and up to Week 48
Change From Baseline in Liver Stiffness as Assessed by Transient Elastography
Liver stiffness was measured using ultrasound elastography. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement.
Measured: Baseline and up to Week 48
Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
TEAEs were defined as adverse events that were reported or worsened on or after the first dose of study treatment. Serious adverse events are adverse events resulting in death, are immediately life threatening, requires in-patient hospitalization or prolongation of existing hospitalization, or results in persistent or significant disability/incapacity.
Measured: Up to Week 48
Change From Baseline in Plasma Levels of Fat-Soluble Vitamin D
Plasma samples were collected to assess levels of fat-soluble vitamins D. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement.
Measured: Baseline and Week 48
Change From Baseline in Plasma Levels of Fat-Soluble Vitamin K
Plasma samples were collected to assess levels of fat-soluble vitamin K. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was computed by subtracting the baseline value from the post-baseline value, with a positive change indicating improvement.
Measured: Baseline and Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2023
- Study start2 Sep 2024
- Primary completion21 Oct 2025
- Study completion21 Oct 2025
Why it stopped: Intercept made a business decision to terminate the study based on FDA's request for voluntary withdrawal of Ocaliva and the issuance of clinical hold on studies under US IND involving OCA.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Biliary AtresiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Intercept Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lynda Szczech, MD, Study director, Intercept Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06121375
- Sponsor's protocol ID
- 747-308
- Status last verified
- Jun 2026
- First submitted
- 2 Nov 2023
- Last updated
- 7 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT06121375
- Acronym / sponsor ID
- 747-308
- Conditions
- Biliary Atresia
- Keywords
- Biliary Atresia, Placebo-controlled, Pharmacokinetics, Pharmacodynamics, Obeticholic Acid, Efficacy, Post-hepatoportoenterostomy
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Intercept made a business decision to terminate the study based on FDA's request for voluntary withdrawal of Ocaliva and the issuance of clinical hold on studies under US IND involving OCA.
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Intercept PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 2 Sep 2024 (actual)
- Primary completion
- 21 Oct 2025 (actual)
- Study completion
- 21 Oct 2025 (actual)
- First posted
- 7 Nov 2023 (actual)
- Results
- Posted 7 Jul 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, care provider)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Participants receiving OCA | Active comparator | Drug: OCAParticipants 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. |
| Participants receiving Matching placebo | Placebo comparator | Drug: Matching PlaceboParticipants 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. |
Who may qualify
- Ages
- 1 day to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (23 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OCAOCA will be administered. | Drug | Participants receiving OCA |
| Matching PlaceboMatching Placebo will be administered. | Drug | Participants receiving Matching placebo |
Primary outcome
Number of Participants With Composite Liver-Related Clinical Events
Time frame: Up to Week 48
Number of participants experiencing any of the following: All-cause death; Liver transplantation; Hospitalization (≥24 hours) for variceal bleeding, hepatic encephalopathy, or spontaneous bacterial peritonitis; Clinically relevant ascites requiring therapeutic paracentesis have been presented. No clinical outcome events were reported at the time of the study termination; hence It was not possible to conduct the planned primary efficacy analysis.
Change From Baseline in Pediatric End-stage Liver Disease (PELD) Score
Time frame: Baseline and up to Week 48
The Pediatric End-stage Liver Disease (PELD) score is a scale used to assess the severity of chronic liver disease in pediatric participants younger than 12 years of age. The total PELD score ranges from negative values to positive values, with no absolute minimum or maximum, with higher scores indicating more severe liver disease and greater urgency for liver transplantation. The score is calculated using total bilirubin, international normalized ratio (INR), albumin, age at listing, and growth failure. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was calculated as the last post-baseline value minus the baseline value. A negative change from baseline indicates improvement, while a positive change indicates worsening.
Change From Baseline in Model of End-stage Liver Disease With Sodium (MELD-NA) Score
Time frame: Baseline and up to Week 48
The Model of End-stage Liver Disease with Sodium (MELD-Na) score is a scale used to assess the severity of chronic liver disease in participants 12 years of age and older, ranging from 6 (less ill) to 40 (gravely ill). Higher scores indicate more severe liver disease and greater urgency for liver transplantation. The score is derived from total bilirubin, serum creatinine, INR, and serum sodium. Baseline was defined as the last assessment prior to first study drug administration. Change from baseline was calculated as the last post-baseline value minus the baseline value. A negative change from baseline indicates improvement, while a positive change indicates worsening.
8 secondary outcomes
Percentage of Participants With Improvement in GGT and Direct Bilirubin (Composite Responder Endpoint)
Time frame: Up to Week 48
Change From Baseline in GGT
Time frame: Baseline and up to Week 48
Change From Baseline in Total and Direct (Conjugated) Bilirubin
Time frame: Baseline and up to Week 48
Change From Baseline in Endogenous Bile Acids
Time frame: Baseline and up to Week 48
Change From Baseline in Liver Stiffness as Assessed by Transient Elastography
Time frame: Baseline and up to Week 48
Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: Up to Week 48
Change From Baseline in Plasma Levels of Fat-Soluble Vitamin D
Time frame: Baseline and Week 48
Change From Baseline in Plasma Levels of Fat-Soluble Vitamin K
Time frame: Baseline and Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Biliary Atresia
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingPhase 3
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
Biliary Atresia
Active, not recruiting
Molecular Characterization for Understanding Biliary Atresia
Biliary Atresia
Active, not recruitingPhase 3
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)
RecruitingPhase 1
Umbilical Cord Mesenchymal Stem Cell Therapy for Biliary Atresia
Biliary Atresia, Kasai Operation
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lynda Szczech, MD study director
Intercept Pharmaceuticals
24 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing OCA in people with Biliary Atresia, run by Intercept Pharmaceuticals at 24 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 7 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in China, Taiwan, Australia, Turkey (Türkiye), Canada and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Jul 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Composite Liver-Related Clinical Events (time frame: Up to Week 48). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2026.