Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
NCT ID: NCT06553768
Last Updated: 2025-12-11
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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RECRUITING
PHASE3
90 participants
INTERVENTIONAL
2024-10-14
2027-02-28
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Maralixibat
Participants will receive maralixibat oral solution 300 μg/kg orally once daily for 1 week and then twice daily for 39 weeks.
Maralixibat
Maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), the study drug (maralixibat) will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of maralixibat. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (at least 16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat.
Placebo
Participants will receive placebo matched to maralixibat oral solution orally once daily for 1 week and then twice daily for 19 weeks. After 20 weeks, participants will receive maralixibat oral solution 300 μg/kg orally once daily for 1 week and then twice daily for 19 weeks.
Placebo
Placebo matched to maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), study drug will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of study drug. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat.
Interventions
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Maralixibat
Maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), the study drug (maralixibat) will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of maralixibat. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (at least 16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat.
Placebo
Placebo matched to maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), study drug will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of study drug. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
1. Diagnosis of ALGS, ICP, PBC, PFIC, or PSC with native liver.
2. Active atopic dermatitis or other non- cholestatic diseases associated with pruritus that are not controlled by standard treatment and that may interfere with the severity assessment of cholestasis-associated pruritus.
3. Decompensated cirrhosis or complications of cirrhosis (e.g., esophageal or gastric variceal bleeding in the last 6 months, high-risk esophageal or gastric varices, ascites, hepatic encephalopathy, hepatorenal syndrome). 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 ≤70,000/mm3. Patients with any condition that further increases bleeding risk are excluded.
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.
6 Months
ALL
No
Sponsors
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Mirum Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Children's Hospital Los Angeles (CHLA)
Los Angeles, California, United States
Stanford Children's Health in Palo Alto
Palo Alto, California, United States
Lurie Children's Hospital
Chicago, Illinois, United States
Ochsner Clinic Foundation
New Orleans, Louisiana, United States
NYU Langone Health
New York, New York, United States
Mount Sinai Hospital
New York, New York, United States
Morgan Stanley Children's Hospital - NewYork Presbyterian
New York, New York, United States
Science 37, Inc (Remote-homebase Telemedicine)
Morrisville, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Baylor College of Medicine
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
Hospital de Criança de Brasília (HCB)
Brasília, , Brazil
Hospital da Criança Santo Antonio
Porto Alegre, , Brazil
Hospital Sírio-Libanês
São Paulo, , Brazil
Stollery Children's Hospital
Edmonton, Alberta, Canada
Hôpital Femme Mère Enfant
Lyon, , France
Hôpitaux Universitaires de Marseille Timone
Marseille, , France
Hôpital Kremlin Bicêtre
Paris, , France
Universitätsklinikum Hamburg Eppendorf - Klinik für Kinder- und Jugendmedizin
Hamburg, , Germany
LMU Klinikum - Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital
Munich, , Germany
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, , Italy
ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione
Palermo, , Italy
Ospedale Pediatrico Bambino Gesu
Rome, , Italy
Hotel Dieu de France
Beirut, , Lebanon
Hospital Infantil de México Federico Gómez
Mexico City, , Mexico
Consultario de Joshue David Covarrubias Esquer
Zapopan, , Mexico
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, , Poland
King Faisal Specialist Hospital & Research Center
Riyadh, , Saudi Arabia
Hospital Sant Joan de Deu
Barcelona, , Spain
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
King's College Hospital NHS Foundation Trust
London, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Lee Bass, MD
Role: primary
Role: primary
Related Links
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Mirum Pharmaceuticals homepage
Other Identifiers
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2024-511287-85-00
Identifier Type: CTIS
Identifier Source: secondary_id
MRX-802
Identifier Type: -
Identifier Source: org_study_id
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