RECRUITING

Evaluation of Maralixibat in Pruritus Associated with General Cholestatic Liver Disease (EXPAND)

Conditions

Cholestatic Liver Disease (except ALGS, PFIC, PBC and PSC)Pruritus Cholestatic Liver Disease Biliary Atresia

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric and adult participants who have cholestatic liver disease with pruritus that has been refractory to other therapies, and who have no other treatment options.

Official Title

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

Quick Facts

Study Start:2024-10-14

Study Completion:2027-02

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06553768

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Months

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	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.

Inclusion Criteria

Exclusion 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.

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.

Contacts and Locations

Study Contact

Clinical Trials Mirum

CONTACT

+16506674085

clinicaltrials@mirumpharma.com

Medinfo Mirum

CONTACT

medinfo@mirumpharma.com

Study Locations (Sites)

Children's Hospital Los Angeles (CHLA)

Los Angeles, California, 90027

United States

Stanford Children's Health in Palo Alto

Palo Alto, California, 94304

United States

Lurie Children's Hospital

Chicago, Illinois, 60611

United States

Ochsner Clinic Foundation

New Orleans, Louisiana, 70121

United States

NYU Langone Health

New York, New York, 10016

United States

Morgan Stanley Children's Hospital - NewYork Presbyterian

New York, New York, 10032

United States

Baylor College of Medicine

Houston, Texas, 77030

United States

University of Utah

Salt Lake City, Utah, 84112

United States

Collaborators and Investigators

Sponsor: Mirum Pharmaceuticals, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-10-14

Study Completion Date2027-02

Study Record Updates

Study Start Date2024-10-14

Study Completion Date2027-02

Terms related to this study

Keywords Provided by Researchers

Pruritus
Cholestatic Liver Disease
Biliary Atresia

Additional Relevant MeSH Terms

Cholestatic Liver Disease (except ALGS, PFIC, PBC and PSC)