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Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

ConditionsAlagille SyndromeProgressive Familial Intrahepatic Cholestasis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorMirum Pharmaceuticals, Inc.

About this trial

The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) treated with Livmarli.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis

Participant prescribed Livmarli

Disqualifiers

Refusal to provide informed consent/assent (if required by the local IRB)

Previously or currently on Livmarli through participation in a clinical study or expanded access program

Participants who have previously received an SBD or LT

Any condition or abnormalities that, in the opinion of the investigator, may interfere with the participant participating in or completing the study

Trial population

All patients with ALGS and PFIC prescribed Livmarli at a participating study center and who consent/assent to participation (if required by the local IRB/IEC) will be included in the study at the discretion of the center investigator.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Livmarli

    Drug

    The recommended dosage is 380 mcg/kg once daily.

  • Livmarli

    Drug

    The recommended dosage us 570 mcg/kg twice daily.

Treatment groups

70 Participants
are divided into 2 treatment groups
Group A: Alagille syndrome (ALGS)1 intervention
Group B: Progressive familial intrahepatic cholestasis (PFIC)1 intervention

Trial outcomes

Primary outcomes

1

Incidence of Long-Term Clinical Outcomes

The dates, and reasons for the following first events (of this first endpoint) will be collected: Surgical Biliary Diversion (SBD), Liver Transplant (LT), and all-cause mortality. In addition, manifestations of clinically evident portal hypertension (CEPH) will be captured during each interval event assessments.

Time frame
Long-term clinical outcomes (SBD, LT, portal hypertension, all-cause mortality) up to 180 days after discontinuation of Livmarli will be recorded.
2

Liver Transplant Indication and Waitlist Status

LT waitlist status will be collected, including when placed on or removed from LT waitlist.

Time frame
LT waitlist status will be collected at enrollment and every 6 months for 5 years.
3

Assessment of Growth and Development

Height and weight will be collected both at the time the participant started Livmarli and at the time of enrollment in the study. Subsequent weight will be collected for up to 5 years. Weight z-score (kilograms) and height z-score (centimeters) will be assessed and reported every year for 5 years.

Time frame
Weight (kilograms) and height (centimeters) z-scores will be collected every year for 5 years.
4

Incidence of Clinical Events Potentially Related to Fat-Soluble Vitamin Deficiencies and Their Long-Term Sequelae

Bleeding events (including all gastrointestinal \[GI\] or non-GI bleeding requiring hospitalization, emergency department care, or transfusion) and fracture events will be reported.

Time frame
The incidence of events will be assessed and reported every year for 5 years.

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Mirum Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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