An Open-label Study to Evaluate the Long-term Safety and Tolerability of LUM001 in Patients With Primary Biliary Cirrhosis

Mirum Pharmaceuticals, Inc.

Overview

Open-label, multicenter study in adults with Primary Biliary Cirrhosis (PBC) designed to evaluate the long-term safety and tolerability of daily dosing with LUM001.

This is an open-label study in subjects with PBC who previously completed the LUM001 201 (CLARITY) study. The study is designed to investigate the long-term safety and tolerability of LUM001 treatment, with daily dosing for up to 2 years. Changes over time, compared to baseline, in liver enzymes, other biochemical markers associated with PBC, pruritus, and other quality of life measures will also be assessed.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Conditions

Primary Biliary Cirrhosis

Interventions

LUM001DRUG

LUM001 administered orally once each day.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Completed a core treatment protocol of LUM001 in the treatment of Primary Biliary Cirrhosis. 2. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures. Exclusion Criteria: 1. Presence of advanced liver disease. 2. History of non-adherence during the subject's participation in the core LUM001 treatment protocol.

Locations (18)

Scripps Clinic

La Jolla, California, United States

University of California at Davis

Sacramento, California, United States

Outcomes

Primary Outcomes

Number of Participants with Adverse Events as a Measure of Safety

Adverse events, serious adverse events, changes in vital signs, laboratory and physical exam findings, and concommitant medication usage from baseline to Week 104

Time frame: 2 years