Primary Biliary Cirrhosis
Conditions
Brief summary
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.
Detailed 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.
Interventions
DRUGLUM001
LUM001 administered orally once each day.
Sponsors
Mirum Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Adverse Events as a Measure of Safety | 2 years | Adverse events, serious adverse events, changes in vital signs, laboratory and physical exam findings, and concommitant medication usage from baseline to Week 104 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 | 2 years | Alkaline phosphatase |
Countries
United Kingdom, United States
Outcome results
None listed