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Ion Transporter Inhibitor

Maralixibat for Cholestatic Liver Disease (RISE Trial)

Phase 2

Waitlist Available

Research Sponsored by Mirum Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of PFIC or ALGS

Be younger than 18 years old

Must not have

History of surgical disruption of the enterohepatic circulation

Predicted complete absence of bile salt excretion pump (BSEP) function

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial will test whether maralixibat is safe and tolerated in young children with ALGS or PFIC.

Who is the study for?

This trial is for infants under 12 months old with a body weight of at least 2.5 kg and diagnosed with cholestatic liver diseases like Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC). Babies should be born after at least 36 weeks of pregnancy, or have reached that postmenstrual age if born earlier. Infants who may need a liver transplant soon, have had certain surgeries on their bile system, or have other significant liver conditions can't participate.

What is being tested?

The study tests the safety and tolerability of maralixibat in young children with ALGS or PFIC. Maralixibat is an investigational drug which means it's still being studied and isn't approved yet for general use.

What are the potential side effects?

While specific side effects for infants taking maralixibat are not detailed here, common side effects in older patients include diarrhea, nausea, abdominal pain, and potential changes in liver enzyme levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with PFIC or ALGS.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I've had surgery that altered my liver and intestine's normal function.

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My body cannot excrete bile salts properly.

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My liver is severely damaged and cannot function properly.

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I have had a liver transplant or may need one soon.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2022 Phase 3 trial • 93 Patients • NCT03905330

57%

Diarrhoea

36%

Pyrexia

21%

Abdominal pain

17%

Rhinorrhoea

15%

Cough

15%

Blood bilirubin increased

13%

Influenza

13%

Alanine aminotransferase increased

11%

Nasopharyngitis

11%

Pruritus

Vitamin D deficiency

Vitamin D decreased

Constipation

Vitamin E decreased

Gastroenteritis

Coronavirus infection

Upper respiratory tract infection

Vomiting

Vitamin E deficiency

Abdominal pain upper

Urinary tract infection

Idiopathic pneumonia syndrome

Cholestasis

International normalised ratio increased

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Maralixibat

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: MaralixibatExperimental Treatment1 Intervention

Participants will receive up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Maralixibat

2015

Completed Phase 3

~260

0 / 5Eligibility criteria met