Biliary AtresiaPhase 2EMBARKNCT04524390
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
A Phase 2 study testing Maralixibat in people with Biliary Atresia, run by Mirum Pharmaceuticals, Inc. at 22 sites in 8 countries.
Trial summary
A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure). Read more about this study
- Who is it for?
- Ages 21 to 111 days
- Conditions the study is for
- Biliary Atresia
- What is being tested?
- Maralixibat
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 75 participants
- Where
- 22 sitesin 8 countries
- Study dates
- 8 Jul 2021to 7 Feb 2024
- Phase
- Phase 2
- 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 21 to 111 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female subjects with body weight ≥2500 g, who are ≥21 days old and <90 days old at the time of HPE (Kasai)
- HPE or Kasai Procedure within 3 weeks prior to randomization
- Clinical diagnosis of biliary atresia
Who cannot join
- Subjects with intractable chronic diarrhea at randomization
- Subjects not tolerating enteral feeds at randomization
- History of ileal resection
- Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
- Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
- Evidence of liver failure (e.g. significant ascites)
Trial locations and contacts
22 sites in 8 countries.
22 sites in 8 countries
United States7 sites
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, 30329
Texas Children's Hospital
Houston, Texas, 77030
NYU Grossman School of Medicine
New York, New York, 10016
New York-Presbyterian - Columbia University Medical Center
New York, New York, 10032
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Division of Gastroenterology & Hepatology
Phoenix, Arizona, 85016
Montefiore Medical Center
The Bronx, New York, 10467
China5 sites
Beijing Pediatric Research Institute
Beijing, Beijing Municipality, 100020
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, 510623
The Children's Hospital, Zhejiang University School of Medicine
Hanzhou, Zhejiang, 310058
Children's hospital of Shanghai
Shanghai, 200062
Children's Hospital of Fudan University
Shanghai, 201102
Vietnam3 sites
Vietnam National Children's Hospital
Hanoi, 115000
Children's Hospital No. 1
Ho Chi Minh City, 740500
Hue Central Hospital
Huế, Thừa Thiên Huế Province
Taiwan2 sites
Taichung Veterans General Hospital
Taichung, 407
Linkou Chang Gung Memorial Hospital
Taoyuan, 333
United Kingdom2 sites
Birmingham Children's Hospital
Birmingham, B4 6NH
King's College Hospital NHS
London
About this study
Official title: Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Biliary Atresia After Hepatoportoenterostomy
A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).
Read the detailed description
This is a double-blind randomized, placebo-controlled study in subjects with Biliary Atresia with a primary endpoint at Week 26 followed by long-term open-label period during which all subjects will receive maralixibat to Week 104.
- Conditions
- Biliary Atresia
- Being tested
- Maralixibat, Placebo
- Keywords
- Biliary Atresia, Kasai, Biliary Tract Diseases, Bile Duct Diseases, Congenital Abnormalities
- Medical subject headings
- Biliary Atresia, Biliary Tract Diseases, Bile Duct Diseases, Congenital Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MaralixibatDrug
Also called Formerly LUM001 and SHP625
A small molecule inhibitor of the ileal bile acid transporter (IBAT)
PlaceboOther
Identical to maralixibat except for the active drug substance
| Study group | Role | What they receive |
|---|---|---|
| Double Blind - Maralixibat | Experimental | The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm. Receives: Drug: Maralixibat |
| Double Blind - Placebo | Placebo comparator | The double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm. Receives: Other: Placebo |
| Open Label - Maralixibat | Experimental | The Open-Label period comprised of 4-8 weeks of dose escalation followed by 70 - 74 weeks of stable dosing treatment. During the OLE, all participants, regardless of treatment assignment in the double-blind period, received maralixibat. Receives: Drug: Maralixibat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change in Total Serum Bilirubin Levels
Measured: From baseline to Week 26
Secondary outcomes (7)
Mean Change in Total Serum Bile Acids
Measured: From baseline to Week 26
Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26
Measured: From baseline to Week 26
Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death.
Liver decompensation (hepatic encephalopathy, variceal bleeding, new persistent ascites)
Measured: From Baseline to Week 26
Proportion of Participants Undergoing Liver Transplantation or Death
Measured: From Baseline to Week 26
Proportion of Participants Observed to Develop Clinically Evident Portal Hypertension Defined as Splenomegaly and Thrombocytopenia (Platelet Count <150 x 109/L) or Clinically Evident Ascites or Endoscopic Evidence of Esophageal or Gastric Varices.
Splenomegaly => (spleen size >2 cm below the costal margin palpated on physical examination)
Measured: From Baseline to Week 26
Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL
Measured: From Baseline to Week 26
Proportion of Participants With Mean sBA Levels ≤40 mmol/L
Measured: From Baseline to Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2020
- Study start8 Jul 2021
- Primary completion7 Nov 2023
- Study completion7 Feb 2024
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.
- More trials run by Mirum Pharmaceuticals, Inc.The sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 75 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04524390
- Sponsor's protocol ID
- MRX-701
- Acronym
- EMBARK
- Status last verified
- Feb 2025
- First submitted
- 20 Aug 2020
- Last updated
- 19 Mar 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Biliary Atresia After Hepatoportoenterostomy
- ClinicalTrials.gov ID
- NCT04524390
- Acronym / sponsor ID
- EMBARK / MRX-701
- Conditions
- Biliary Atresia
- Keywords
- Biliary Atresia, Kasai, Biliary Tract Diseases, Bile Duct Diseases, Congenital Abnormalities
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 8 Jul 2021 (actual)
- Primary completion
- 7 Nov 2023 (actual)
- Study completion
- 7 Feb 2024 (actual)
- First posted
- 24 Aug 2020 (actual)
- Results
- Posted 17 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).
Detailed description
This is a double-blind randomized, placebo-controlled study in subjects with Biliary Atresia with a primary endpoint at Week 26 followed by long-term open-label period during which all subjects will receive maralixibat to Week 104.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Double Blind - Maralixibat | Experimental | Drug: MaralixibatThe double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm. |
| Double Blind - Placebo | Placebo comparator | Other: PlaceboThe double-blind period comprised of 4-8 weeks of dose escalation followed by 18 - 22 weeks of stable dosing treatment, after which participants were transferred to the open-label arm. |
| Open Label - Maralixibat | Experimental | Drug: MaralixibatThe Open-Label period comprised of 4-8 weeks of dose escalation followed by 70 - 74 weeks of stable dosing treatment. During the OLE, all participants, regardless of treatment assignment in the double-blind period, received maralixibat. |
Who may qualify
- Ages
- 21 to 111 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Male or female subjects with body weight ≥2500 g, who are ≥21 days old and <90 days old at the time of HPE (Kasai)
- HPE or Kasai Procedure within 3 weeks prior to randomization
- Clinical diagnosis of biliary atresia
Key exclusion criteria (6 listed)
- Subjects with intractable chronic diarrhea at randomization
- Subjects not tolerating enteral feeds at randomization
- History of ileal resection
- Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
- Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
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 |
|---|---|---|
| MaralixibatAlso: Formerly LUM001 and SHP625A small molecule inhibitor of the ileal bile acid transporter (IBAT) | Drug | Double Blind - Maralixibat, Open Label - Maralixibat |
| PlaceboIdentical to maralixibat except for the active drug substance | Other | Double Blind - Placebo |
Primary outcome
Mean Change in Total Serum Bilirubin Levels
Time frame: From baseline to Week 26
7 secondary outcomes
Mean Change in Total Serum Bile Acids
Time frame: From baseline to Week 26
Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26
Time frame: From baseline to Week 26
Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death.
Time frame: From Baseline to Week 26
Proportion of Participants Undergoing Liver Transplantation or Death
Time frame: From Baseline to Week 26
Proportion of Participants Observed to Develop Clinically Evident Portal Hypertension Defined as Splenomegaly and Thrombocytopenia (Platelet Count <150 x 109/L) or Clinically Evident Ascites or Endoscopic Evidence of Esophageal or Gastric Varices.
Time frame: From Baseline to Week 26
Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL
Time frame: From Baseline to Week 26
Proportion of Participants With Mean sBA Levels ≤40 mmol/L
Time frame: From Baseline to Week 26
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Mar 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
The record lists no contact. Use the official record to reach the sponsor.
22 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Maralixibat in people with Biliary Atresia, run by Mirum Pharmaceuticals, Inc. at 22 sites in 8 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 6 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: finished, as of the sponsor's last update on 19 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, China, Vietnam, Taiwan, United Kingdom and 3 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 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.
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 17 Feb 2025). 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?
Mean Change in Total Serum Bilirubin Levels (time frame: From baseline to Week 26). 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 19 Mar 2025.