Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

Study Overview
=================
Brief Summary
-----------------
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.

Official Title
-----------------
The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease

Conditions
-----------------
Alagille Syndrome

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


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Diagnosis of Alagille Syndrome Evidence of cholestasis Moderate to severe pruritus Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures Exclusion Criteria: Surgical disruption of the enterohepatic circulation Liver transplant History or presence of other concomitant liver disease Females who are pregnant or lactating Known HIV infection

Ages Eligible for Study
-----------------
Minimum Age: 12 Months
Maximum Age: 18 Years

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

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

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

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: LUM001<br>LUM001 for oral administration | Drug: LUM001<br>* LUM001 administered orally<br>|
| Placebo Comparator: Placebo<br>Placebo administered orally once each day | Drug: Placebo<br>* Placebo administered orally<br>|

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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus | Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period. | Baseline, Week 13/Early Termination | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level | Fasting sBA level was measured by using a liquid chromatography mass spectrometry method. | Baseline, Week 13/Early Termination | 
| Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels | Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here. | Baseline, Week 13/Early Termination | 
| Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations | Liver enzyme levels of total bilirubin and direct bilirubin were reported here. | Baseline, Week 13/Early Termination |