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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04524390
Recruitment Status : Active, not recruiting
First Posted : August 24, 2020
Last Update Posted : May 31, 2023
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

Study Description
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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).

Condition or disease Intervention/treatment Phase
Biliary Atresia Drug: Maralixibat Other: Placebo Phase 2

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.
Study Design
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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

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Mean change in total serum bilirubin levels [ Time Frame: From baseline to Week 26 ]

Secondary Outcome Measures :
  1. Mean change in total serum bile acids [ Time Frame: From baseline to Week 26 ]
  2. Proportion of participants with total bilirubin levels <2 mg/dL at Week 26 [ Time Frame: At Week 26 ]
  3. Time to liver transplantation or death [ Time Frame: From Baseline to Week 26 ]
  4. Proportion of participants undergoing liver transplantation or death [ Time Frame: From Baseline to Week 26 ]
  5. Proportion of participants with liver-related clinical event, including liver transplantation, liver decompensation and death [ Time Frame: From Baseline to Week 26 ]
  6. Mean change in serum alanine aminotransferase (ALT) [ Time Frame: From Baseline to Week 26 ]
  7. Mean change in serum γ-glutamyltransferase (GGT) [ Time Frame: From Baseline to Week 26 ]
  8. Mean change in blood platelets [ Time Frame: From Baseline to Week 26 ]
  9. Mean change in serum albumin [ Time Frame: From Baseline to Week 26 ]

Eligibility Criteria
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Ages Eligible for Study:   21 Days to 111 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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)
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
More Information
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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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Mirum Pharmaceuticals, Inc.:
Biliary Atresia
Kasai
Biliary Tract Diseases
Bile Duct Diseases
Congenital Abnormalities
Additional relevant MeSH terms:
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Biliary Atresia
Bile Duct Diseases
Biliary Tract Diseases
 Digestive System Diseases
Digestive System Abnormalities
Congenital Abnormalities