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OpenTrials
Completed

NCT Number: NCT04185363

An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.

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Key information

Conditions

Progressive Familial Intrahepatic Cholestasis (PFIC) Bile Duct Diseases Biliary Tract Diseases Cholestasis Cholestasis, progressive familial intrahepatic 1 Digestive System Diseases Liver Diseases

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hospital Italiano de Buenos Aires, Buenos Aires, Argentina

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About this study

The study will be conducted at multiple sites in North America, Europe, Asia, and South America.

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
  • Completion of study MRX-502

Exclusion criteria

  • Any female who is pregnant or lactating or who is planning to become pregnant
  • Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0)
  • History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment
  • Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat
  • Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study
  • Cognitive impairment of the subject or caregiver that would, in the opinion of the investigator, preclude appropriate understanding of study information and compliance with study procedures

Treatment and study plan

Maralixibat

Drug

All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) twice daily

Primary outcomes

  1. Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Severity Score

    Time frame: From Baseline to Weeks 75-78

    The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)

Secondary outcomes

  1. Maintenance of ItchRO(Obs) Response (Weeks 15 - 26)

    Time frame: Week 15 - 26

    Maintenance of treatment effect is defined as the proportion of participants in the MRX-MRX treatment group (MRX-502 and MRX-503 data for subjects on MRX in the MRX-502 study) who obtain an ItchRO (Obs) response from Week 15 to Week 26 in Study MRX-503 in the primary cohort and PFIC cohort.

  2. Proportion of ItchRO(Obs) Responders Over Time

    Time frame: Baseline to EOT

    Proportion of ItchRO responders over time at each study visit using the 4-week study period prior to the visit as per ItchRO(Obs) responder definition.

  3. Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Frequency Score

    Time frame: From Baseline to Weeks 75-78

    The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)

  4. Mean Change From Baseline Over Time in Total Serum Bile Acid (sBA) Levels

    Time frame: Baseline to week 70

    Mean change from baseline over time in total serum bile acid (sBA) levels

  5. Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26

    Time frame: From Week 18 to Week 26

    Proportion of subjects who experience an sBA control over time from Week 18 to Week 26

  6. Change From Baseline in Height Z-score

    Time frame: From baseline to Week 70

    Height-for-age Z-score measures a participant's height relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate height above the reference population mean, and negative Z-scores indicate height below the reference population mean. Higher Z-scores generally indicate greater linear growth relative to the reference population. Change from baseline was calculated as post-baseline height-for-age Z-score minus baseline height-for-age Z-score.

  7. Change From Baseline in Weight Z-score

    Time frame: From Baseline to Week 70

    Weight-for-age Z-score measures a participant's weight relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate weight above the reference population mean, and negative Z-scores indicate weight below the reference population mean. Higher Z-scores generally indicate greater body weight relative to the reference population. Change from baseline was calculated as post-baseline weight-for-age Z-score minus baseline weight-for-age Z-score.

Sponsors and collaborators

Lead sponsor

Mirum Pharmaceuticals, Inc.

Industry

Registry information

Official study title

An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

Important dates

Study start
2020
Primary completion
2025
Study completion
2025
First posted
Dec 4, 2019
Registry last updated
Jun 23, 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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