Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (EMBARK)
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ClinicalTrials.gov Identifier: NCT04524390 |
Recruitment Status :
Active, not recruiting
First Posted : August 24, 2020
Last Update Posted : May 31, 2023
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Condition or disease | Intervention/treatment | Phase |
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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 : | August 2023 |
Estimated Study Completion Date : | March 2025 |

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.
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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 ]

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Ages Eligible for Study: | 21 Days 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

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: | May 31, 2023 |
Last Verified: | May 2023 |
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 |