Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (EMBARK)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04524390 |
Recruitment Status :
Recruiting
First Posted : August 24, 2020
Last Update Posted : April 22, 2022
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Biliary Atresia | Drug: Maralixibat Other: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 72 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | July 8, 2021 |
Estimated Primary Completion Date : | February 2023 |
Estimated Study Completion Date : | August 2024 |

Arm | Intervention/treatment |
---|---|
Experimental: Maralixibat
Maralixibat chloride oral solution administered twice daily, up to 600* microgram per kilogram, for 26 weeks and in the OLE for all patients. *equivalent to 570 mcg/kg/day maralixibat free base |
Drug: Maralixibat
A small molecule inhibitor of the ileal bile acid transporter (IBAT)
Other Name: Formerly LUM001 and SHP625 |
Placebo Comparator: Placebo
Placebo oral solution for 26 weeks. All placebo participants who complete Week 26 and continue in the open label extension (OLE) will receive maralixibat after Week 26.
|
Other: Placebo
Identical to maralixibat except for the active drug substance |
- Mean change in total serum bilirubin levels [ Time Frame: From baseline to Week 26 ]
- Mean change in total serum bile acids [ Time Frame: From baseline to Week 26 ]
- Proportion of participants with total bilirubin levels <2 mg/dL at Week 26 [ Time Frame: At Week 26 ]
- Time to liver transplantation 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 with liver-related clinical event, including liver transplantation, liver decompensation and death [ Time Frame: From Baseline to Week 26 ]
- Mean change in serum alanine aminotransferase (ALT) [ Time Frame: From Baseline to Week 26 ]
- Mean change in serum γ-glutamyltransferase (GGT) [ Time Frame: From Baseline to Week 26 ]
- Mean change in blood platelets [ Time Frame: From Baseline to Week 26 ]
- Mean change in serum albumin [ Time Frame: From Baseline to Week 26 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | up to 111 Days (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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)

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04524390
Contact: Clinical Trials Mirum | +16506674085 | clinicaltrials@mirumpharma.com | |
Contact: Medinfo Mirum | medinfo@mirumpharma.com |
United States, District of Columbia | |
Medstar Georgetown University Hospital | Recruiting |
Washington, District of Columbia, United States, 20007-2113 | |
Contact: Carissa Vinovskis 202-444-3133 carissa.vinovskis@medstar.net | |
Principal Investigator: Udeme Ekong, MD | |
United States, Louisiana | |
Ochsner Clinic Foundation | Recruiting |
New Orleans, Louisiana, United States, 70121 | |
Contact: Ryan Himes 504-842-3000 ryan.himes@ochsner.org | |
Principal Investigator: Ryan Himes, MD | |
United States, New York | |
Columbia University Irving Medical Center | Recruiting |
New York, New York, United States, 10032 | |
Contact: Bo Lu 212-305-3009 Bl2699@cumc.columbia.edu | |
Principal Investigator: Jennifer Vittorio, MD | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | Recruiting |
Philadelphia, Pennsylvania, United States, 19104 | |
Contact: Kathleen Loomes 267-426-7223 LOOMES@email.chop.edu | |
Principal Investigator: Kathleen Loomes, MD | |
UPMC Children's Hospital of Pittsburgh | Recruiting |
Pittsburgh, Pennsylvania, United States, 15224 | |
Contact howerd@upmc.edu | |
Principal Investigator: Simon Horslen, MD | |
United States, Texas | |
Texas Children's Hospital | Recruiting |
Houston, Texas, United States, 77030 | |
Contact: Stanley Oseghale 832-822-3565 sxosegha@texaschildrens.org | |
Principal Investigator: Anna Banc-Husu, MD | |
China, Beijing | |
Beijing Pediatric Research Institute | Recruiting |
Beijing, Beijing, China, 100020 | |
Contact: Yingxia Zhang 86-17731110041 | |
Principal Investigator: Long Li, MD | |
China, Guangdong | |
Guangzhou Women and Children's Medical Center | Recruiting |
Guangzhou, Guangdong, China, 510623 | |
Contact: Yufeng Feng 86-15989212597 | |
Principal Investigator: Jiakang Yu, MD | |
Germany | |
Hannover Medical School | Recruiting |
Hanover, Germany | |
Principal Investigator: Ulrich Baumann, MD | |
Poland | |
Instytut Pomnik-Centrum Zdrowia Dziecka | Recruiting |
Warsaw, Poland | |
Contact: Sylwia Cichosz +48 22 815 70 42 s.cichosz@ipczd.pl | |
Principal Investigator: Irena Jankowska, MD | |
United Kingdom | |
King's College Hospital NHS | Recruiting |
London, United Kingdom | |
Contact: Sophie Cant sophie.cant1@nhs.net | |
Principal Investigator: Vandana Jain, MBBCh |
Responsible Party: | Mirum Pharmaceuticals, Inc. |
ClinicalTrials.gov Identifier: | NCT04524390 |
Other Study ID Numbers: |
MRX-701 |
First Posted: | August 24, 2020 Key Record Dates |
Last Update Posted: | April 22, 2022 |
Last Verified: | April 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Biliary Atresia Kasai Biliary Tract Diseases Bile Duct Diseases Congenital Abnormalities |
Biliary Atresia Bile Duct Diseases Biliary Tract Diseases |
Digestive System Diseases Digestive System Abnormalities Congenital Abnormalities |