A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
NCT ID: NCT04729751
Last Updated: 2025-02-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
27 participants
INTERVENTIONAL
2021-09-09
2024-12-17
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Maralixibat
Participants will receive up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable.
Maralixibat
Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL)
* 400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base
* 600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base
Interventions
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Maralixibat
Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL)
* 400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base
* 600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. \<12 months of age at the baseline visit (ROW). \>31 days and \<12 months of age at the baseline visit (US).
3. Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.
4. Diagnosis of PFIC or ALGS
Exclusion Criteria
2. History of surgical disruption of the enterohepatic circulation
3. History of liver transplant or imminent need for liver transplant
4. Decompensated cirrhosis
5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion
6. Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study
0 Days
364 Days
ALL
No
Sponsors
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Mirum Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Children Hospital LA
Los Angeles, California, United States
University of California - San Francisco
San Francisco, California, United States
Medstar Georgetown University Hospital
Washington D.C., District of Columbia, United States
Ochsner Hospital for Children
New Orleans, Louisiana, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Cliniques Universitaires Saint-Luc
Brussels, , Belgium
Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês
São Paulo, , Brazil
Hôpital Kremlin Bicêtre
Le Kremlin-Bicêtre, , France
Hopital Necker
Paris, , France
Consultorio de Joshue David Covarrubias Esquer
Zapopan, , Mexico
Instytut Pomnik-Centrum Zdrowia Dziecka
Warsaw, , Poland
King's College Hospital
London, , United Kingdom
Countries
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Related Links
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Genetics Home Reference - PFIC
Mirum Pharmaceuticals homepage
Genetics Home Reference - ALGS
US FDA Resources
Other Identifiers
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2020-004628-40
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
MRX-801
Identifier Type: -
Identifier Source: org_study_id
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