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

Phase 4RecruitingNCT07290257

Mirum Pharmaceuticals, Inc.100 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

OTHER

Masking

NONE

Enrollment

100

Conditions

Alagille Syndrome

Interventions

Livmarli Oral ProductDRUG

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

Eligibility

Sex: ALLMin age: 2 Months

Medical Language ↔ Plain English

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

Locations (13)

Cliniques Universitaires Saint Luc (UCLouvain)

Brussels, Belgium

NOT_YET_RECRUITING

University Hospital Gent (UZ Gent)

Ghent, Belgium

Outcomes

Primary Outcomes

Adverse Events (AEs)

Number and proportion of participants with AEs.

Time frame: Up to 7 days after the last dose of Livmarli.

Liver function tests (LFTs) - measuring enzyme activity or concentration

Change in LFTs from Baseline: Total and direct bilirubin, aminotransferases, GGT, and alkaline phosphatase.

Time frame: Once every 3-4 months, for 5 years.

Fat-Soluble Vitamins (FSV) Concentration

Change in FSV levels (vitamins A, D, and E) from Baseline.

Time frame: Once every 3-4 months, for 5 years.

International Normalized Ratio (INR)

Change in INR level from Baseline.

Time frame: Once every 3-4 months, for 5 years.

Long-Term Clinical Outcomes

Number of participants with surgical biliary diversion, liver transplantation, liver transplant waitlist status change, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, liver decompensation, and death.

Time frame: Once every 3-4 months, for 5 years.

Central Contacts

Clinical Trials Mirum

CONTACT

+16506674085 clinicaltrials@mirumpharma.com

Data from ClinicalTrials.gov

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