Maralixibat for Cholestasis

Recruiting at 29 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: Mirum Pharmaceuticals, Inc.

Disqualifiers: Pregnancy, Lactation, Non-compliance, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

90 Participants Needed

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Maralixibat oral solution

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive Maralixibat to evaluate long-term safety and efficacy

Long-term

Treatment Details

Interventions

• Maralixibat

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

Who Is Running the Clinical Trial?

Mirum Pharmaceuticals, Inc.

Lead Sponsor

Trials

32

Recruited

102,000+