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

Mirum Pharmaceuticals, Inc.3 sites in 1 country20 target enrollmentAugust 2013

ConditionsAlagille Syndrome

InterventionsLUM001 Placebo

DrugsLUM001 Placebo

Overview

Phase: Phase 2
Intervention: LUM001
Conditions: Alagille Syndrome
Sponsor: Mirum Pharmaceuticals, Inc.
Enrollment: 20
Locations: 3
Primary Endpoint: Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level
Status: Completed
Last Updated: 7 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

August 2013

End Date

March 2015

Last Updated

7 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Mirum Pharmaceuticals, Inc.

Responsible Party

Sponsor

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

Arms & Interventions

LUM001

LUM001 administered orally once each day

Intervention: LUM001

Placebo

Placebo administered orally once each day

Intervention: Placebo

Outcomes

Primary Outcomes

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

Time Frame: 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 Outcomes

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