A Multicentre Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001 Also Known as Maralixibat (MRX), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

Mirum Pharmaceuticals, Inc.3 sites in 1 country19 target enrollmentDecember 20, 2013

ConditionsAlagille Syndrome

InterventionsLUM001 (Maralixibat)

DrugsLUM001 (Maralixibat)

Overview

Phase: Phase 2
Intervention: LUM001 (Maralixibat)
Conditions: Alagille Syndrome
Sponsor: Mirum Pharmaceuticals, Inc.
Enrollment: 19
Locations: 3
Primary Endpoint: Change From MRX Baseline to Week 48 in Fasting sBA Levels
Status: Completed
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of this extension study is to determine the long-term safety and tolerability of an investigational treatment (LUM001 also known as Maralixibat) in children with ALGS who have completed participation in a core LUM001 treatment protocol. Efficacy will be assessed by evaluating the effect of LUM001 on pruritus, biochemical markers of pruritus, as well as biochemical markers of cholestasis and liver disease.

Registry

clinicaltrials.gov

Start Date

December 20, 2013

End Date

June 17, 2020

Last Updated

4 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Mirum Pharmaceuticals, Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arms & Interventions

LUM001 (Maralixibat)

LUM001 also known as Maralixibat (MRX) administered orally up to twice each day

Intervention: LUM001 (Maralixibat)

Outcomes

Primary Outcomes

Change From MRX Baseline to Week 48 in Fasting sBA Levels

Time Frame: MRX baseline to Week 48

The primary endpoint of this study was the mean change from MRX baseline to Week 48 in fasting sBA level.

Secondary Outcomes

Change From MRX Baseline Over Time in Fasting sBA Levels(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
Change From MRX Baseline to Week 48 in Clinician Xanthoma Severity Score(MRX baseline to Week 48)
Change From MRX Baseline Over Time in Clinician Xanthoma Severity Score(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
Change From MRX Baseline to Week 48 in Pruritus(MRX baseline to Week 48)
Change From MRX Baseline Over Time in Pruritus(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
Secondary: Change From MRX Baseline to Week 48 in Alkaline Phosphatase(MRX baseline to Week 48)
Change From MRX Baseline Over Time in Alkaline Phosphatase(MRX baseline to end of treatment (maximum exposure was 336 weeks))
Change From MRX Baseline Over Time in Alanine Aminotransferase(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
Change From MRX Baseline to Week 48 in Aspartate Aminotransferase(MRX baseline to Week 48)
Change From MRX Baseline Over Time in Aspartate Aminotransferase(MRX baseline to End of treatment (maximum exposure was 336 weeks))
Change From MRX Baseline to Week 48 in Gamma Glutamyltransferase(MRX baseline to Week 48)
Change From MRX Baseline Over Time in Gamma Glutamyltransferase(MRX baseline to End of Treatment (maximum exposure was 336 weeks))
Change From MRX Baseline to Week 48 in Alanine Aminotransferase(MRX baseline to Week 48)
Change From MRX Baseline to Week 48 in Total and Direct Bilirubin(MRX baseline to Week 48)
Change From MRX Baseline Over Time in Total and Direct Bilirubin(MRX baseline to End of Treatment (maximum exposure was 336 weeks))