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Evaluating maralixibat for itching in cholestatic liver disease

Randomized Double-Blind Placebo-Controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Participants With Cholestatic Pruritus

Phase 3 Interventional Mirum Pharmaceuticals, Inc. · NCT06553768

This study is testing a new medication called maralixibat to see if it can help reduce itching in adults and children with cholestatic liver disease who haven't found relief from other treatments.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment90 (estimated)
Ages6 Months and up
SexAll
SponsorMirum Pharmaceuticals, Inc. Industry-sponsored
Locations32 sites (Los Angeles, California and 31 other locations)
Trial IDNCT06553768 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and effectiveness of maralixibat in treating pruritus associated with cholestatic liver disease in both pediatric and adult patients who have not responded to other treatments. Participants will be monitored over a period of time to evaluate the drug's impact on their symptoms. The study will take place across multiple sites in North America, Europe, the Middle East, and South America, ensuring a diverse participant pool. Both maralixibat and a placebo will be administered to compare outcomes effectively.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 6 months and older diagnosed with cholestatic liver disease and experiencing refractory pruritus.

Not a fit: Patients with cholestatic liver disease who do not experience pruritus or those who have other treatment options available may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly alleviate itching for patients suffering from cholestatic liver disease.

How similar studies have performed: While there have been studies on cholestatic liver disease, the specific use of maralixibat for pruritus in this context is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Informed consent and assent (as applicable)
2. Age ≥6 months at time of baseline visit
3. Diagnosis of cholestatic liver disease with cholestatic pruritus based on the presence of chronic liver biochemical abnormalities (\>90 days) and/or pathological evidence of progressive liver disease.
4. If taking antipruritics or ursodeoxycholic acid, the participant has to be on a stable dosing regimen (i.e., same dose and frequency in the 30 days prior to the screening visit and will continue this dosing regimen up to Week 40 \[adjustment for body weight is allowed\]).
5. Access to email or telephone for scheduled participant contacts and access to smart phone or tablet for PROs.
6. Ability to read and/or understand the questionnaires (both caregivers and participants ≥9 years of age).
7. For participants ≤18 years of age: Access to consistent caregiver(s) during the study.

Exclusion Criteria:

Those who meet any of the following criteria are NOT eligible to participate in the study:

1. Diagnosis of ALGS, ICP, PBC, PFIC, or PSC with native liver.
2. Current or recent history (\<1 year) of atopic dermatitis or other non-cholestatic diseases associated with pruritus.
3. History of decompensated cirrhosis or complications of cirrhosis (e.g., esophageal/gastric varices, ascites, hepatic encephalopathy, hepatorenal syndrome). In patients who have had a liver transplant, this exclusion criterion applies to the post-transplant period only. Patients with compensated cirrhosis with preserved hepatic synthetic function and absence of complications are eligible.
4. Suspected or proven cholangiocarcinoma or hepatocellular carcinoma.
5. Unstable and/or serious medical disease that is likely to impair the ability to participate in all aspects of the study, confound efficacy and/or safety assessments, or result in substantially shortened life expectancy (e.g., any active malignancy including hematological malignancy, end-stage heart failure, active infection, acute and chronic diarrhea). Exceptionally, previous history of malignancy, adequately treated/in remission, that in opinion of investigator and medical monitor does not impact participant safety and participation in the study, may be allowed.
6. Laboratory results during the screening visit as follows:

   1. Platelet count ≤150,000/mm3
   2. Albumin \<30 g/L
   3. INR ≥1.5 (after intravenous or subcutaneous supplementation of vitamin K)
   4. Total bilirubin \>10 mg/dL
   5. ALT \>10× ULN
7. Use of an IBAT inhibitor within 8 weeks prior to the screening visit.
8. Known intolerance/hypersensitivity to maralixibat or its excipients.
9. History of nonadherence to medical regimens, unreliability, medical condition, mental instability, or cognitive impairment that, in the opinion of the investigator, could compromise the validity of informed consent, compromise the safety of the participant, or lead to nonadherence with the study protocol or inability to conduct the study procedures.

Where this trial is running

Los Angeles, California and 31 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Cholestatic Liver DiseasePruritusBiliary Atresia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.