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)

Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age2+
SponsorMirum Pharmaceuticals, Inc.

About this trial

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.

Eligibility criteria

Qualifiers

Understand and execute an Informed consent and assent (as applicable)

For participants with ALGS ≥2 months of age at Day 1

For participants with PFIC, ≥3 months of age at Day 1

A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis, or a clinically and/or genetically confirmed PFIC diagnosis

Disqualifiers

History of Liver Transplant

Any Livmarli contraindications (as per SmPC)

Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study

Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded-access programs is acceptable.)

Trial design

Treatments tested in this trial

  • Livmarli Oral Product

Treatment groups

230 Participants
are divided into 1 treatment group

Sponsors and collaborators