Safety and Efficacy Study of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Alagille Syndrome

Phase 2CompletedNCT01903460

Mirum Pharmaceuticals, Inc.20 enrolled

Overview

The study is a randomized, double-blind, placebo-controlled study to evaluate the safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 versus placebo on the biochemical markers and pruritus associated with Alagille Syndrome.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Alagille Syndrome

Interventions

LUM001DRUG

PlaceboDRUG

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 (3)

Birmingham Children's Hospital

Birmingham, West Midlands, United Kingdom

Leeds Teaching Hospitals

Leeds, West Yorkshire, United Kingdom

Kings College Hospital

London, United Kingdom

Outcomes

Primary Outcomes

Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level

Participants were required to fast for at least 4 hours; only water was permitted prior to collection. A negative change from baseline indicates that the level of bile acid decreased.

Time frame: Baseline to 13 weeks or end of treatment

Secondary Outcomes

Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes

Analysis of liver enzymes included alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP). A negative change from baseline indicates that the level of that enzyme decreased.

Time frame: Baseline to 13 weeks or end of treatment

Change From Baseline to Week 13 (End of Treatment) in Pruritus as Measured by The Patient And Observer Itch Reported Outcome (ItchRO) Average Daily Scores

The ItchRO was administered as a twice daily electronic diary (eDiary). Children ≥9 years of age completed the patient ItchRO; those between the ages of 5 and 8 completed the patient ItchRO with the assistance of their caregiver. There was no patient report for subjects under the age of 5. ItchRO scores range from 0 to 4, with the higher score indicating increasing itch severity. ItchRO average daily scores were calculated as the sum of daily scores (ie, the maximum of morning and evening scores) divided by the number of days. The average daily score was calculated by using the 7 days pre-treatment for baseline, and the last 7 days of treatment for Week 13. A negative change from Baseline indicates that itch severity decreased.

Time frame: Baseline to 13 weeks or end of treatment

Data from ClinicalTrials.gov

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