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Completed

NCT Number: NCT04524390

Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai

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).

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Key information

Conditions

Biliary Atresia Bile Duct Diseases Biliary Tract Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Abnormalities Digestive System Diseases

Age range

21 day–111 day

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Beijing Pediatric Research Institute, Beijing, Beijing Municipality, China

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Maralixibat

Drug

A small molecule inhibitor of the ileal bile acid transporter (IBAT)

Other names: Formerly LUM001 and SHP625

Placebo

Other

Identical to maralixibat except for the active drug substance

Primary outcomes

  1. Mean Change in Total Serum Bilirubin Levels

    Time frame: From baseline to Week 26

Secondary outcomes

  1. Mean Change in Total Serum Bile Acids

    Time frame: From baseline to Week 26

  2. Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26

    Time frame: From baseline to Week 26

  3. 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

    Liver decompensation (hepatic encephalopathy, variceal bleeding, new persistent ascites)

  4. Proportion of Participants Undergoing Liver Transplantation or Death

    Time frame: From Baseline to Week 26

  5. 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

    Splenomegaly => (spleen size >2 cm below the costal margin palpated on physical examination)

  6. Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL

    Time frame: From Baseline to Week 26

  7. Proportion of Participants With Mean sBA Levels ≤40 mmol/L

    Time frame: From Baseline to Week 26

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Registry information

Official study 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

Acronym: EMBARK

Important dates

Study start
2021
Primary completion
2023
Study completion
2024
First posted
Aug 24, 2020
Registry last updated
Mar 19, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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