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

NCT04168385 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: MRX-800
• Study Type: interventional
• Target: 52
• Locations: 8 countries
• Sites: 17

Brief 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.

Conditions

Cholestatic Liver Disease

Keywords

PFIC; ALGS; Maralixibat; Bile Duct Diseases; Liver Diseases; Biliary Tract Diseases; Digestive System Diseases; Pediatric

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events

  TEAE = Treatment-emergent Adverse Event; AESI = Adverse Event of Special Interest..

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

Secondary Outcomes (2)

• Long-Term Effect on Pruritus

  From Baseline through Week 160, including Change from Baseline values.

• Long-Term Effect on Serum Bile Acid Levels

  From Baseline through Week 160, including Change from Baseline values.

Study Arms (1)

Maralixibat

EXPERIMENTAL

Participants will all receive Maralixibat oral solution

Drug: Maralixibat

Interventions

Maralixibat DRUG

Maralixibat chloride oral solution orally twice daily (up to 1200\* mcg/kg/day), and according to indication. \*equivalent to 1140 mcg/kg/day maralixibat

Also known as: Formerly LUM001 and SHP625

Maralixibat

Eligibility Criteria

• Age: 12 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• Having completed the EOT Visit, for subjects coming from the maralixibat Phase 2 studies.
• Having completed the entire duration of the study (i.e., core and extension, if applicable), for subjects coming from the maralixibat Phase 3 studies.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Mirum Pharmaceuticals, Inc.: lead

Study Sites (17)

Children'S Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Riley Hospital For Children

Indianapolis, Indiana, 46202, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Baylor College of Medicine/Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Children's Hospital Westmead

Westmead, New South Wales, 2145, Australia

Location

The Royal Children'S Hospital Melbourne

Parkville, Victoria, 3052, Australia

Location

Cliniques Universitaires Saint-Luc

Brussels, Belgium

Location

Hospital for Sick Children

Toronto, Canada

Location

Hopital Necker-Enfants Malades

Paris, 75015, France

Location

Hopital Kremlin Bicetre

Paris, 94275, France

Location

The Children's Memorial Health Institute

Warsaw, 04-730, Poland

Location

Hospital Universitario La Paz- Hospital Materno Infantil

Madrid, 261, Spain

Location

Birmingham Children's Hospital

Birmingham, United Kingdom

Location

Leeds Teaching Hospital NHS Trust

Leeds, LS1 3EX, United Kingdom

Location

Paediatric Liver Center, Kings College Hospital

London, SE5 9RS, United Kingdom

Location

Related Links

• Genetics Home Reference - PFIC
• Genetics Home Reference - Alagille syndrome
• US FDA Resources
• Mirum Pharmaceuticals homepage

MeSH Terms

Conditions

Bile Duct Diseases; Liver Diseases; Biliary Tract Diseases; Digestive System Diseases

Interventions

maralixibat

Results Point of Contact

• Title: Mirum Clinical Trials
• Organization: Mirum Pharmaceuticals, Inc.

medinfo@mirumpharma.com

16506674085

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 1, 2019
• First Posted: November 19, 2019
• Study Start: January 16, 2020
• Primary Completion: September 4, 2024
• Study Completion: September 4, 2024
• Last Updated: December 8, 2025
• Results First Posted: December 8, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (6); AU (2); GB (3); BE (1); ES (1); FR (2); CA (1); PL (1)