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

Mirum Pharmaceuticals

Status and phase: Enrolling; Phase 4

Conditions: Alagille Syndrome

Treatments: Drug: Livmarli Oral Product

Study type: Interventional

Funder types: Industry

Identifiers

NCT07290257

MRX-803

Details and patient eligibility

About

The key objectives of this low-intervention clinical study are to evaluate tolerability, long-term safety and long-term efficacy for patients with Alagille syndrome (ALGS) who are prescribed Livmarli.

Full description

This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with Alagille syndrome (ALGS) who are treated with Livmarli for cholestatic pruritus.

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

Enrollment: 100 estimated patients

Sex: All

Ages: 2+ months old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Informed consent and assent (as applicable)
• ≥2 months of age at Day 1
• A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis
• For the primary cohort, prescribed Livmarli at time of study entry
• For the supplemental cohort, prescribed Livmarli prior to study entry

Exclusion criteria

• History of Liver Transplant
• Any contraindications against Livmarli (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.)
• Baseline data before start of treatment of Livmarli are unavailable (<2 values before treatment) for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameter

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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100 participants in 1 patient group

Patients with Alagile Syndrome treated with Livmarli

Experimental group

Description:

Primary cohort with a minimum of 45 previously untreated (treatment naïve). Supplemental cohort of participants who have previously received Livmarli.

Treatment:

Drug: Livmarli Oral Product