Maralixibat for Cholestasis

Recruiting in Palo Alto (17 mi)

+29 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Mirum Pharmaceuticals, Inc.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Breakthrough Therapy

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial aims to see if maralixibat, a medication, is safe and can be tolerated by patients over time. It involves patients who have already been using the medication. The study is being conducted at various locations around the world.

Eligibility Criteria

This trial is for patients with Progressive Familial Intrahepatic Cholestasis who have completed the MRX-502 study. They must be able to give informed consent and not have any health issues or lab results that could make the trial unsafe for them. Pregnant or breastfeeding women, those planning pregnancy, or anyone with a history of non-compliance in the previous study cannot participate.

Inclusion Criteria

Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)

Completion of study MRX-502

Exclusion Criteria

I stopped taking maralixibat due to a serious side effect in the MRX-502 study.

I have always followed medical advice and participated reliably in past studies.

You have any other medical conditions or abnormal test results that could make it unsafe for you to participate in the study or affect your ability to complete it.

Treatment Details

Interventions

• Maralixibat (IBAT Inhibitor)

Trial Overview The trial is testing Maralixibat's long-term safety and effectiveness in treating cholestasis conditions like PFIC. It's an open label extension, meaning everyone knows they're getting Maralixibat and there’s no placebo group.

Participant Groups

Group I: Maralixibat Experimental Treatment 1 Intervention

All subjects will receive Maralixibat oral solution

Maralixibat is already approved in United States, European Union, Canada for the following indications:

🇺🇸 Approved in United States as Livmarli for:

• Treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older
• Treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 3 months of age and older

🇪🇺 Approved in European Union as Livmarli for:

• Treatment of cholestatic pruritus in patients with Alagille syndrome

🇨🇦 Approved in Canada as Livmarli for:

• Treatment of cholestatic pruritus in adults with Alagille syndrome

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

University of Alberta, Edmonton Clinic Health Academy Edmonton, Canada

University of Texas, Health Science Center San Antonio San Antonio, TX

University of Texas Southwestern Medical Center Dallas, TX

Cincinnati Children's Hospital Cincinnati, OH

More Trial Locations

Who Is Running the Clinical Trial?

Mirum Pharmaceuticals, Inc. Lead Sponsor

References