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

Phase 2

Completed

• Conditions: Alagille Syndrome
• Interventions: Drug: LUM001; Drug: Placebo

• Registration Number: NCT01903460
• Lead Sponsor: Mirum Pharmaceuticals, Inc.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-07-16
• Study First Submitted QC: 2013-07-16
• Study First Posted: 2013-07-19
• Study Start: 2013-08
• Primary Completion: 2015-02
• Study Completion: 2015-03
• Results First Submitted: 2015-11-02
• Results First Submitted QC: 2015-11-02
• Results First Posted: 2015-12-07
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-15
• Last Update Posted: 2019-03-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
LUM001	LUM001	LUM001 administered orally once each day
Placebo	Placebo	Placebo administered orally once each day

Primary Outcome Measures

Name	Time	Method
Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level	Baseline to 13 weeks or end of treatment	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.

Secondary Outcome Measures

Name	Time	Method
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	Baseline to 13 weeks or end of treatment	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.
Change From Baseline to Week 13 (End of Treatment) in Liver Enzymes	Baseline to 13 weeks or end of treatment	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.

Trial Locations

Locations (3)

Kings College Hospital; 🇬🇧 London, United Kingdom

Birmingham Children's Hospital; 🇬🇧 Birmingham, West Midlands, United Kingdom

Leeds Teaching Hospitals; 🇬🇧 Leeds, West Yorkshire, United Kingdom