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

Study Overview
=================
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.

Official Title
-----------------
An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety and Tolerability of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Primary Biliary Cirrhosis

Conditions
-----------------
Primary Biliary Cirrhosis

Intervention / Treatment
-----------------
* Drug: LUM001


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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: LUM001<br>LUM001 administered orally once each day. | Drug: LUM001<br>* LUM001 administered orally once each day.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 | Alkaline phosphatase | 2 years | 
| Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 | Liver enzymes (ALT, AST) | 2 years | 
| Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 | Fasting serum bile acid | 2 years | 
| Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 | C4 | 2 years | 
| Efficacy analysis as changes in liver enzymes and other biochemical markers from baseline to Week 104 | Quality of life assessments (itch and sleep scores) | 2 years |