Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
EMBARK
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
1 other identifier
interventional
75
8 countries
22
Brief 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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Jul 2021
22 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 20, 2020
CompletedFirst Posted
Study publicly available on registry
August 24, 2020
CompletedStudy Start
First participant enrolled
July 8, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 7, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
February 7, 2024
CompletedResults Posted
Study results publicly available
February 17, 2025
CompletedMarch 19, 2025
February 1, 2025
2.3 years
August 20, 2020
December 20, 2024
March 10, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Mean Change in Total Serum Bilirubin Levels
From baseline to Week 26
Secondary Outcomes (7)
Mean Change in Total Serum Bile Acids
From baseline to Week 26
Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26
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.
From Baseline to Week 26
Proportion of Participants Undergoing Liver Transplantation or Death
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.
From Baseline to Week 26
- +2 more secondary outcomes
Study Arms (3)
Double Blind - Maralixibat
EXPERIMENTALThe 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 COMPARATORThe 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
EXPERIMENTALThe 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.
Interventions
A small molecule inhibitor of the ileal bile acid transporter (IBAT)
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (22)
Phoenix Children's Division of Gastroenterology & Hepatology
Phoenix, Arizona, 85016, United States
Children's Healthcare of Atlanta - Emory University School of Medicine
Atlanta, Georgia, 30329, United States
NYU Grossman School of Medicine
New York, New York, 10016, United States
New York-Presbyterian - Columbia University Medical Center
New York, New York, 10032, United States
Montefiore Medical Center
The Bronx, New York, 10467, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Beijing Pediatric Research Institute
Beijing, Beijing Municipality, 100020, China
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, 510623, China
The Children's Hospital, Zhejiang University School of Medicine
Hanzhou, Zhejiang, 310058, China
Children's hospital of Shanghai
Shanghai, 200062, China
Children's Hospital of Fudan University
Shanghai, 201102, China
Hannover Medical School
Hanover, Germany
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, Poland
KK women's and Children's hospital
Bukit Timah, 229899, Singapore
Taichung Veterans General Hospital
Taichung, 407, Taiwan
Linkou Chang Gung Memorial Hospital
Taoyuan District, 333, Taiwan
Birmingham Children's Hospital
Birmingham, B4 6NH, United Kingdom
King's College Hospital NHS
London, United Kingdom
Hue Central Hospital
Huế, Thừa Thiên Huế Province, Vietnam
Vietnam National Children's Hospital
Hanoi, 115000, Vietnam
Children's Hospital No. 1
Ho Chi Minh City, 740500, Vietnam
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Mirum Clinical Trials
- Organization
- Mirum Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 20, 2020
First Posted
August 24, 2020
Study Start
July 8, 2021
Primary Completion
November 7, 2023
Study Completion
February 7, 2024
Last Updated
March 19, 2025
Results First Posted
February 17, 2025
Record last verified: 2025-02