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Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai (EMBARK)

Primary Purpose

Biliary Atresia

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Maralixibat
Placebo
Sponsored by
Mirum Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Biliary Atresia focused on measuring Biliary Atresia, Kasai, Biliary Tract Diseases, Bile Duct Diseases, Congenital Abnormalities

Eligibility Criteria

21 Days - 111 Days (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male or female subjects with body weight ≥2500 g, who are ≥21 days old and <90 days old at the time of HPE (Kasai)
  2. HPE or Kasai Procedure within 3 weeks prior to randomization
  3. Clinical diagnosis of biliary atresia

Exclusion Criteria:

  1. Subjects with intractable chronic diarrhea at randomization
  2. Subjects not tolerating enteral feeds at randomization
  3. History of ileal resection
  4. Diagnosis of biliary atresia splenic malformation syndrome or cystic biliary atresia
  5. Evidence of another non-biliary atresia pathology involving the intrahepatic bile duct (e.g., paucity, sclerosing cholangitis)
  6. Evidence of liver failure (e.g. significant ascites)

Sites / Locations

  • Phoenix Children's Division of Gastroenterology & Hepatology
  • Standford Medicine Childrens Health
  • Medstar Georgetown University Hospital
  • Kidz Medical
  • Children's Healthcare of Atlanta - Emory University School of Medicine
  • The University of Chicago Medical Center
  • Ochsner Clinic Foundation
  • University of Nebraska Medical Center
  • Columbia University Irving Medical Center
  • New York-Presbyterian - Columbia University Medical Center
  • Northwell Health System
  • Children's Hospital of Philadelphia
  • UPMC Children's Hospital of Pittsburgh
  • Le Bonheur Children's Hospital
  • Texas Children's Hospital
  • University Texas Health Science Center
  • Seattle Children's Hospital
  • Beijing Pediatric Research Institute
  • Guangzhou Women and Children's Medical Center
  • The Children's Hospital, Zhejiang University School of Medicine
  • Children's hospital of Shanghai
  • Children's Hospital of Fudan University
  • Universitätsklinikum Essen
  • Hannover Medical School
  • The University of Hong Kong Queen Mary Hospital
  • Aziendo Ospedaliera papa Giovanni XXIIII
  • Instytut Pomnik-Centrum Zdrowia Dziecka
  • KK women's and Children's hospital
  • Taichung Veterans General Hospital
  • National Taiwan University Hospital
  • Linkou Chang Gung Memorial Hospital
  • Birmingham Children's Hospital
  • King's College Hospital NHS
  • Hue Central Hospital
  • Vietnam National Children's Hospital
  • Children's Hospital No. 1

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Maralixibat

Placebo

Arm Description

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

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.

Outcomes

Primary Outcome Measures

Mean change in total serum bilirubin levels

Secondary Outcome Measures

Mean change in total serum bile acids
Proportion of participants with total bilirubin levels <2 mg/dL at Week 26
Time to liver transplantation or death
Proportion of participants undergoing liver transplantation or death
Proportion of participants with liver-related clinical event, including liver transplantation, liver decompensation and death
Mean change in serum alanine aminotransferase (ALT)
Mean change in serum γ-glutamyltransferase (GGT)
Mean change in blood platelets
Mean change in serum albumin

Full Information

First Posted
August 20, 2020
Last Updated
October 5, 2023
Sponsor
Mirum Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04524390
Brief Title
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
Acronym
EMBARK
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 8, 2021 (Actual)
Primary Completion Date
November 2023 (Anticipated)
Study Completion Date
March 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mirum Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Biliary Atresia
Keywords
Biliary Atresia, Kasai, Biliary Tract Diseases, Bile Duct Diseases, Congenital Abnormalities

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
72 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Maralixibat
Arm Type
Experimental
Arm Description
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
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Maralixibat
Other Intervention Name(s)
Formerly LUM001 and SHP625
Intervention Description
A small molecule inhibitor of the ileal bile acid transporter (IBAT)
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Identical to maralixibat except for the active drug substance
Primary Outcome Measure Information:
Title
Mean change in total serum bilirubin levels
Time Frame
From baseline to Week 26
Secondary Outcome Measure Information:
Title
Mean change in total serum bile acids
Time Frame
From baseline to Week 26
Title
Proportion of participants with total bilirubin levels <2 mg/dL at Week 26
Time Frame
At Week 26
Title
Time to liver transplantation or death
Time Frame
From Baseline to Week 26
Title
Proportion of participants undergoing liver transplantation or death
Time Frame
From Baseline to Week 26
Title
Proportion of participants with liver-related clinical event, including liver transplantation, liver decompensation and death
Time Frame
From Baseline to Week 26
Title
Mean change in serum alanine aminotransferase (ALT)
Time Frame
From Baseline to Week 26
Title
Mean change in serum γ-glutamyltransferase (GGT)
Time Frame
From Baseline to Week 26
Title
Mean change in blood platelets
Time Frame
From Baseline to Week 26
Title
Mean change in serum albumin
Time Frame
From Baseline to Week 26

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Days
Maximum Age & Unit of Time
111 Days
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Facility Information:
Facility Name
Phoenix Children's Division of Gastroenterology & Hepatology
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Standford Medicine Childrens Health
City
Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Medstar Georgetown University Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007-2113
Country
United States
Facility Name
Kidz Medical
City
Miramar
State/Province
Florida
ZIP/Postal Code
33025
Country
United States
Facility Name
Children's Healthcare of Atlanta - Emory University School of Medicine
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
The University of Chicago Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
Ochsner Clinic Foundation
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70121
Country
United States
Facility Name
University of Nebraska Medical Center
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68114
Country
United States
Facility Name
Columbia University Irving Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
New York-Presbyterian - Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Northwell Health System
City
New York
State/Province
New York
ZIP/Postal Code
11042
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Le Bonheur Children's Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38103
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University Texas Health Science Center
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Beijing Pediatric Research Institute
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100020
Country
China
Facility Name
Guangzhou Women and Children's Medical Center
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510623
Country
China
Facility Name
The Children's Hospital, Zhejiang University School of Medicine
City
Hanzhou
State/Province
Zhejiang
ZIP/Postal Code
310058
Country
China
Facility Name
Children's hospital of Shanghai
City
Shanghai
ZIP/Postal Code
200062
Country
China
Facility Name
Children's Hospital of Fudan University
City
Shanghai
ZIP/Postal Code
201102
Country
China
Facility Name
Universitätsklinikum Essen
City
Essen
ZIP/Postal Code
45147
Country
Germany
Facility Name
Hannover Medical School
City
Hanover
Country
Germany
Facility Name
The University of Hong Kong Queen Mary Hospital
City
Hong Kong
ZIP/Postal Code
0000
Country
Hong Kong
Facility Name
Aziendo Ospedaliera papa Giovanni XXIIII
City
Bergamo
ZIP/Postal Code
24127
Country
Italy
Facility Name
Instytut Pomnik-Centrum Zdrowia Dziecka
City
Warsaw
Country
Poland
Facility Name
KK women's and Children's hospital
City
Bukit Timah
ZIP/Postal Code
229899
Country
Singapore
Facility Name
Taichung Veterans General Hospital
City
Taichung
ZIP/Postal Code
407
Country
Taiwan
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
Linkou Chang Gung Memorial Hospital
City
Taoyuan
ZIP/Postal Code
333
Country
Taiwan
Facility Name
Birmingham Children's Hospital
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Name
King's College Hospital NHS
City
London
Country
United Kingdom
Facility Name
Hue Central Hospital
City
Huế
State/Province
Thừa Thiên Huế
Country
Vietnam
Facility Name
Vietnam National Children's Hospital
City
Hanoi
ZIP/Postal Code
115000
Country
Vietnam
Facility Name
Children's Hospital No. 1
City
Ho Chi Minh City
ZIP/Postal Code
740500
Country
Vietnam

12. IPD Sharing Statement

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Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai

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