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NCT Number: NCT04729751

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).

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].

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Key information

Conditions

Progressive Familial Intrahepatic Cholestasis Abnormalities, Multiple Alagille Syndrome Bile Duct Diseases Biliary Tract Diseases Cardiovascular Abnormalities Cardiovascular Diseases Cholestasis Cholestasis, Intrahepatic Cholestasis, progressive familial intrahepatic 1 Cholestatic Liver Disease Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Heart Defects, Congenital Liver Diseases

Age range

0 day–364 day

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Cliniques Universitaires Saint-Luc, Brussels, Belgium

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Body weight of ≥2.5 kg
  • <12 months of age at the baseline visit (ROW). >31 days and <12 months of age at the baseline visit (US).
  • 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.
  • Diagnosis of PFIC or ALGS

Exclusion criteria

  • Predicted complete absence of bile salt excretion pump (BSEP) function
  • History of surgical disruption of the enterohepatic circulation
  • History of liver transplant or imminent need for liver transplant
  • Decompensated cirrhosis
  • 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
  • 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

Treatment and study plan

Maralixibat

Drug

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

Primary outcomes

  1. Frequency of Treatment-emergent Adverse Events [TEAEs]

    Time frame: From Baseline through to Week 13

    TEAEs = Treatment-emergent Adverse Events

Secondary outcomes

  1. Change in Fasting Serum Bile Acid (sBA) Levels

    Time frame: From Baseline through to Week 13

    sBA = serum bile acid

  2. To Evaluate the Effect on Liver Enzymes (ALT)

    Time frame: From Baseline through to Week 13

    ALT= alanine aminotransferase.

  3. Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin E

    Time frame: From Baseline through to Week 13

    Change from baseline to Week 13 in vitamin E.

  4. Maximum Level of Maralixibat Concentration in Plasma From Baseline to Week 13 for ALGS

    Time frame: At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit

    BID=twice daily; QD=once daily.

    Systemic concentrations of maralixibat in plasma were determined at the following visits:

    • Approximately 2.5 hours after the morning dose at Week 0/Day 1 of dosing
    • Before dosing and ~2.5 hours after the morning dose at Week 6, Week 10, and Week 13

    Stable dosing occurred at 400 μg/kg QD for ALGS or at the highest tolerated dose.

    Note: The values added in section Measured Values are for maralixibat dose of 400 µg/kg QD.

  5. To Evaluate the Effect on Liver Enzymes (AST)

    Time frame: From Baseline through to Week 13.

    AST= aspartate aminotransferase

  6. To Evaluate the Effect on Bilirubin

    Time frame: From Baseline through to Week 13

    Bilirubin

  7. Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin A

    Time frame: From Baseline through to Week 13

    Change from baseline to Week 13 in vitamin A

  8. Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamins D and K

    Time frame: From Baseline through to Week 13

    Change from baseline to Week 13 in vitamins D and K.

  9. Maximum Level of Maralixibat Concentration in Plasma From Baseline to Week 13 for PFIC

    Time frame: At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit

    BID=twice daily; QD=once daily.

    Systemic concentrations of maralixibat in plasma were determined at the following visits:

    • Approximately 2.5 hours after the morning dose at Week 0/Day 1 of dosing
    • Before dosing and ~2.5 hours after the morning dose at Week 6, Week 10, and Week 13

    Stable dosing occurred at 300 μg/kg QD, 300 μg/kg BID and 600 μg/kg BID for PFIC or at the highest tolerated dose.

    Note: The values added in section Measured Values are for maralixibat dose of 600 µg/kg BID.

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Acronym: RISE

Important dates

Study start
2021
Primary completion
2024
Study completion
2024
First posted
Jan 28, 2021
Registry last updated
Jun 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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