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). (RISE)

  • STATUS
    Recruiting
  • End date
    Aug 20, 2023
  • participants needed
    12
  • sponsor
    Mirum Pharmaceuticals, Inc.
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Updated on 4 October 2022
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Summary

This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].

Description

This is an open label study where all participants will receive maralixibat treatment.

Details
Condition Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease
Treatment Maralixibat
Clinical Study IdentifierNCT04729751
SponsorMirum Pharmaceuticals, Inc.
Last Modified on4 October 2022

Eligibility

 Yes No Not Sure

Inclusion Criteria

Body weight of ≥2.5 kg
<12 months of age at the baseline visit (ROW). >31 days and <12 months of age at the baseline visit (US)
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
Diagnosis of PFIC or ALGS

Exclusion Criteria

Predicted complete absence of bile salt excretion pump (BSEP) function
History of surgical disruption of the enterohepatic circulation
History of liver transplant or imminent need for liver transplant
Decompensated cirrhosis
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
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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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