Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

Phase 2CompletedNCT02057692

Mirum Pharmaceuticals, Inc.37 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Alagille Syndrome

Interventions

LUM001DRUG

LUM001 administered orally

PlaceboDRUG

Placebo administered orally

Eligibility

Sex: ALLMin age: 12 MonthsMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Diagnosis of Alagille Syndrome 2. Evidence of cholestasis 3. Moderate to severe pruritus 4. Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures Exclusion Criteria: 1. Surgical disruption of the enterohepatic circulation 2. Liver transplant 3. History or presence of other concomitant liver disease 4. Females who are pregnant or lactating 5. Known HIV infection

Locations (13)

Children's Hospital Los Angeles

Los Angeles, California, United States

University of California at San Francisco

San Francisco, California, United States

Children's Hospital Colorado

Outcomes

Primary Outcomes

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.

Time frame: Baseline, Week 13/Early Termination

Secondary Outcomes

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.

Time frame: 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.

Time frame: 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.

Time frame: Baseline, Week 13/Early Termination

Data from ClinicalTrials.gov

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