Health Trials

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

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 design

Treatments tested in this trial

  • Livmarli
  • Livmarli

Treatment groups

70 Participants
are divided into 2 treatment groups

Sponsors and collaborators

Mirum Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov