CompletedPhase 2

MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study

United States52 participantsStarted 2020-01-16

Plain-language summary

Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia.

Who can participate

Age range12 Months

SexALL

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Inclusion criteria

✓. Provide informed consent and assent (as applicable) per the Institutional Review Board/Ethics Committee (IRB/EC).
✓. Previously participated in a maralixibat study and with approval of the Medical Monitor. Previous participation is defined as:
✓. At least 1 year of age
✓. Males, and females of non-childbearing potential. Males and non-pregnant, non-lactating females of childbearing potential who are sexually active must agree to use acceptable contraception during the study and 30 days following the last dose of the study medication. Females of childbearing potential must have a negative pregnancy test.
✓. Caregivers (and/or age appropriate subjects) must have access to email or phone for scheduled remote visits if applicable.
✓. Subject and caregiver willingness to comply with all study visits and requirements.

Exclusion criteria

✕. Experienced an AE or SAE related to maralixibat during the lead-in protocol that led to permanent discontinuation of the subject from maralixibat.
✕. Any conditions or abnormalities (including laboratory abnormalities) which, in the opinion of the Investigator or Medical Monitor may compromise the safety of the subject or interfere with the subject participating in or completing the study.
✕. History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator or Sponsor medical monitor, could compromise the validity of informed consent, compromise the safety of the subject, or lead to non-adherence with the study protocol or inability to conduct the study procedures.

What they're measuring

Incidence of Treatment-Emergent Adverse Events

Timeframe: From informed consent through approximately 4.5 years, including 30 days after last dose.

Trial details

NCT IDNCT04168385

SponsorMirum Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-09-04

Results submitted2025-09-03

View on ClinicalTrials.gov More Cholestatic Liver Disease trials