NCT02160782

Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)

Long-Term, Open-Label Study With a Double-Blind, Placebo-Controlled, Randomized Drug Withdrawal Period of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Alagille Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: Mirum Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with Alagille Syndrome (ALGS) designed to evaluate the safety and efficacy of LUM001 (Also known as maralixibat or MRX).

Detailed description

The study is divided into 6 parts: a 6-week open-label, dose escalation period, a 12-week open-label stable dosing period, a 4-week randomized, double-blind, placebo-controlled drug withdrawal period, a 26-week long-term stable dosing period, and an a 52-week optional follow-up treatment period, and a long-term optional follow-up treatment period for eligible participants who choose to stay on treatment with LUM001.

Conditions

• Alagille Syndrome

Interventions

Type	Name	Description
DRUG	LUM001 (Maralixibat)	LUM001, also known as Maralixibat (MRX) will be administered orally Once Daily (OD). To be administered Twice Daily (BID) for patients who are eligible.
DRUG	Placebo	Placebo will be administered orally once daily during randomized withdrawal period

Timeline

• Start date: 2014-10-28
• Primary completion: 2020-05-28
• Completion: 2020-05-28
• First posted: 2014-06-11
• Last updated: 2021-07-14
• Results posted: 2021-07-14

Locations

9 sites across 6 countries: Australia, Belgium, France, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study