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Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)

NCT07290257 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 230

Last updated 2026-06-23

No results posted yet for this study

Summary

In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment.

In patients with Progressive Familial Intrahepatic Cholestasis (PFIC), the key objectives are to evaluate tolerability, long-term safety (including possible liver toxicity and potential impact of chronic exposure to propylene glycol (PG)), long-term efficacy, and growth and development.

Conditions

  • Alagille Syndrome
  • Progressive Familial Intrahepatic Cholestasis

Interventions

DRUG

Livmarli Oral Product

Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.

Sponsors & Collaborators

  • Mirum Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-09-25
Primary Completion
2030-09-22
Completion
2030-12-22

Countries

  • Belgium
  • France
  • Germany
  • Greece
  • Italy
  • Netherlands
  • Portugal
  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07290257 on ClinicalTrials.gov