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

Alagille Syndrome Clinical Trial

— IMAGO  

Official title:

A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Paediatric Patients With Alagille Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01903460
• Other study ID #: LUM001-302
• Secondary ID: 2012-005346-38SH
• Status: Completed
• Phase: Phase 2
• Start date: August 2013
• Est. completion date: March 2015

Study information

• Verified date: March 2019
• Source: Mirum Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: March 2015
• Est. primary completion date: February 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Months to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Alagille Syndrome
• Cholestasis
• Liver Diseases
• Syndrome

Intervention

Drug:
• LUM001

• Placebo

Locations

Country	Name	City	State
United Kingdom	Birmingham Children's Hospital	Birmingham	West Midlands
United Kingdom	Leeds Teaching Hospitals	Leeds	West Yorkshire
United Kingdom	Kings College Hospital	London

Sponsors (1)

Lead Sponsor	Collaborator
Mirum Pharmaceuticals, Inc.

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline to 13 weeks or end of treatment
Secondary	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.	Baseline to 13 weeks or end of treatment
Secondary	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.	Baseline to 13 weeks or end of treatment