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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04524390
Recruitment Status : Recruiting
First Posted : August 24, 2020
Last Update Posted : April 22, 2022
Sponsor:
Information provided by (Responsible Party):
Mirum Pharmaceuticals, Inc.

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.

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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 : February 2023
Estimated Study Completion Date : August 2024

Resource links provided by the National Library of Medicine


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




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 ]


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Ages Eligible for Study:   up 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)

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


Contacts
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Contact: Clinical Trials Mirum +16506674085 clinicaltrials@mirumpharma.com
Contact: Medinfo Mirum medinfo@mirumpharma.com

Locations
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United States, District of Columbia
Medstar Georgetown University Hospital Recruiting
Washington, District of Columbia, United States, 20007-2113
Contact: Carissa Vinovskis    202-444-3133    carissa.vinovskis@medstar.net   
Principal Investigator: Udeme Ekong, MD         
United States, Louisiana
Ochsner Clinic Foundation Recruiting
New Orleans, Louisiana, United States, 70121
Contact: Ryan Himes    504-842-3000    ryan.himes@ochsner.org   
Principal Investigator: Ryan Himes, MD         
United States, New York
Columbia University Irving Medical Center Recruiting
New York, New York, United States, 10032
Contact: Bo Lu    212-305-3009    Bl2699@cumc.columbia.edu   
Principal Investigator: Jennifer Vittorio, MD         
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Kathleen Loomes    267-426-7223    LOOMES@email.chop.edu   
Principal Investigator: Kathleen Loomes, MD         
UPMC Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact       howerd@upmc.edu   
Principal Investigator: Simon Horslen, MD         
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Stanley Oseghale    832-822-3565    sxosegha@texaschildrens.org   
Principal Investigator: Anna Banc-Husu, MD         
China, Beijing
Beijing Pediatric Research Institute Recruiting
Beijing, Beijing, China, 100020
Contact: Yingxia Zhang    86-17731110041      
Principal Investigator: Long Li, MD         
China, Guangdong
Guangzhou Women and Children's Medical Center Recruiting
Guangzhou, Guangdong, China, 510623
Contact: Yufeng Feng    86-15989212597      
Principal Investigator: Jiakang Yu, MD         
Germany
Hannover Medical School Recruiting
Hanover, Germany
Principal Investigator: Ulrich Baumann, MD         
Poland
Instytut Pomnik-Centrum Zdrowia Dziecka Recruiting
Warsaw, Poland
Contact: Sylwia Cichosz    +48 22 815 70 42    s.cichosz@ipczd.pl   
Principal Investigator: Irena Jankowska, MD         
United Kingdom
King's College Hospital NHS Recruiting
London, United Kingdom
Contact: Sophie Cant       sophie.cant1@nhs.net   
Principal Investigator: Vandana Jain, MBBCh         
Sponsors and Collaborators
Mirum Pharmaceuticals, Inc.
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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: April 22, 2022
Last Verified: April 2022

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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