A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). (RISE)

March 14, 2024 updated by: Mirum Pharmaceuticals, Inc.

Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome

This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

This is an open label study where all participants will receive maralixibat treatment.

Study Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Belgium
      • Brussels, Belgium
        • Cliniques Universitaires Saint-Luc
  • Brazil
      • São Paulo, Brazil, 01308-000
        • Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês
  • France
      • Le Kremlin-Bicêtre, France
        • Hopital Kremlin Bicetre
      • Paris, France
        • Hôpital Necker
  • Mexico
      • Zapopan, Mexico, 45050
        • Consultorio de Joshue David Covarrubias Esquer
  • Poland
      • Warsaw, Poland
        • Instytut Pomnik-Centrum Zdrowia Dziecka
  • United Kingdom
      • London, United Kingdom
        • King's College Hospital
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Children Hospital LA
      • San Francisco, California, United States, 94158
        • University of California - San Francisco
    • District of Columbia
      • Washington, District of Columbia, United States, 20007
        • MedStar Georgetown University Hospital
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • Ochsner Hospital for Children
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh
    • Texas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

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.

Eligibility Criteria

Ages Eligible for Study

1 second to 11 months (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Body weight of ≥2.5 kg
  2. <12 months of age at the baseline visit (ROW). >31 days and <12 months of age at the baseline visit (US).
  3. Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.
  4. Diagnosis of PFIC or ALGS

Exclusion criteria:

  1. Predicted complete absence of bile salt excretion pump (BSEP) function
  2. History of surgical disruption of the enterohepatic circulation
  3. History of liver transplant or imminent need for liver transplant
  4. Decompensated cirrhosis
  5. Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion
  6. Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Maralixibat
Participants will receive up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable.
Drug: Maralixibat

Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL)

  • 400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base
  • 600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base
Other Names:
  • Formerly LUM001 and SHP625

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Frequency of treatment-emergent adverse events [TEAEs]
Time Frame: From Baseline through to Week 13
From Baseline through to Week 13

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in fasting serum bile acid (sBA) levels
Time Frame: From Baseline through to Week 13
From Baseline through to Week 13
To evaluate the effect on liver enzymes (ALT, AST) and bilirubin
Time Frame: From Baseline through to Week 13
From Baseline through to Week 13
To evaluate the effect on LSVs
Time Frame: From Baseline through to Week 13
From Baseline through to Week 13
To assess the plasma level of maralixibat in infant participants
Time Frame: At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit
At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mirum Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

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 Major Dates

Study Start (Actual)

September 9, 2021

Primary Completion (Actual)

August 22, 2023

Study Completion (Estimated)

December 1, 2024

Study Registration Dates

First Submitted

January 25, 2021

First Submitted That Met QC Criteria

January 27, 2021

First Posted (Actual)

January 28, 2021

Study Record Updates

Last Update Posted (Actual)

March 15, 2024

Last Update Submitted That Met QC Criteria

March 14, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MRX-801
  • 2020-004628-40 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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