NCT06553768

Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)

Randomized Double-Blind Placebo-Controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Participants With Cholestatic Pruritus

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 90 (estimated)
• Sponsor: Mirum Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 6 Months
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric and adult participants who have cholestatic liver disease with pruritus that has been refractory to other therapies, and who have no other treatment options.

Detailed description

This study will be conducted in multiple sites in North America, Europe, Middle East and South America.

Conditions

• Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)

Interventions

Type	Name	Description
DRUG	Maralixibat	Maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), the study drug (maralixibat) will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of maralixibat. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (at least 16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat.
OTHER	Placebo	Placebo matched to maralixibat will be provided as an oral solution along with 0.5-, 1.0-, and 3.0-mL sized dosing dispensers. During the double-blind dose escalation period (4 weeks), study drug will be administered once daily for 1 week and then twice daily (BID; morning and evening). During the double-blind stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the double-blind dose-escalation period) of study drug. During the open-label dose escalation period (4 weeks), all participants will receive maralixibat treatment once daily for 1 week and then twice daily (BID; morning and evening). During the open-label stable dosing period (16 weeks), participants will be treated with 300 μg/kg BID or the maximum tolerated dose (determined during the open-label dose-escalation period) of maralixibat.

Timeline

• Start date: 2024-10-14
• Primary completion: 2026-10-01
• Completion: 2027-02-01
• First posted: 2024-08-14
• Last updated: 2026-03-30

Locations

24 sites across 10 countries: United States, Brazil, Canada, France, Germany, Italy, Lebanon, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study