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

Mirum Pharmaceuticals

Status and phase: Withdrawn; Phase 2

Conditions: Primary Biliary Cirrhosis

Treatments: Drug: LUM001

Study type: Interventional

Funder types: Industry

Identifiers

NCT02321306

LUM001-202

2014-003433-26 (EudraCT Number)

Details and patient eligibility

About

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.

Full description

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.

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

LUM001

Experimental group

Description:

LUM001 administered orally once each day.

Treatment:

Drug: LUM001