← Back to Search

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at baseline, week 6, week 10, week 13 or early termination visit

Awards & highlights

RISE Trial 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.Check my eligibility

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.See study design

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.

RISE Trial Eligibility Criteria

Inclusion Criteria

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

Select...

I have been diagnosed with PFIC or ALGS.

RISE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at baseline, week 6, week 10, week 13 or early termination visit

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at baseline, week 6, week 10, week 13 or early termination visit

This trial's timeline: 3 weeks for screening, Varies for treatment, and at baseline, week 6, week 10, week 13 or early termination visit for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Frequency of treatment-emergent adverse events [TEAEs]

Secondary outcome measures

Change in fasting serum bile acid (sBA) levels

To assess the plasma level of maralixibat in infant participants

To evaluate the effect on LSVs

+1 more

Side effects data

From 2022 Phase 3 trial • 93 Patients • NCT03905330

57%

Diarrhoea

36%

Pyrexia

21%

Abdominal pain

17%

Rhinorrhoea

15%

Blood bilirubin increased

15%

Cough

13%

Influenza

13%

Alanine aminotransferase increased

11%

Nasopharyngitis

11%

Pruritus

Vitamin E decreased

Constipation

Vitamin D deficiency

Vitamin D decreased

Vitamin E deficiency

Gastroenteritis

Coronavirus infection

Upper respiratory tract infection

Vomiting

Urinary tract infection

Abdominal pain upper

International normalised ratio increased

Cholestasis

Idiopathic pneumonia syndrome

100%

80%

60%

40%

20%

Study treatment Arm

Maralixibat

Placebo

RISE 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 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Mirum Pharmaceuticals, Inc.Lead Sponsor

29 Previous Clinical Trials

1,634 Total Patients Enrolled

1 Trials studying Cholestatic Liver Disease

52 Patients Enrolled for Cholestatic Liver Disease

Media Library

Maralixibat (Ion Transporter Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04729751 — Phase 2

Cholestatic Liver Disease Research Study Groups: Maralixibat

Cholestatic Liver Disease Clinical Trial 2023: Maralixibat Highlights & Side Effects. Trial Name: NCT04729751 — Phase 2

Maralixibat (Ion Transporter Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04729751 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are any new participants being accepted for this investigation?

"Affirmative. According to the information on clinicaltrials.gov, this experiment is presently in search of participants and opened for enrollment since September 9th 2021. As of July 18th 2022, 12 individuals are expected from 7 different medical centres."

Answered by AI

Does this research include adults aged 45 and above?

"Participants in this research must have a minimal age of 0 Days and be younger than 364 days."

Answered by AI

What is the current enrolment rate for this clinical trial?

"This trial necessitates 12 eligible participants; those who fulfil the criteria for inclusion. The research extends to Children Hospital LA in Los Angeles and Children's Hospital of Pittsburgh in Pennsylvania."

Answered by AI

Has Maralixibat earned the approval of the US Food and Drug Administration?

"Taking into account the current level of research, Maralixibat was deemed a 2 on our safety scale as there is some evidence that it can be used safely but no clinical trials have been done to verify its efficacy."

Answered by AI

Does this medical research project possess any novel characteristics?

"Since its inception in 2019, Maralixibat has been studied extensively. The pioneering trial was sponsored by Mirum Pharmaceuticals and included 52 participants. This initial study led to the drug receiving Phase 2 approval from regulators, with 4 ongoing studies hosted across 26 cities and 20 countries worldwide today."

Answered by AI

Who is the ideal participant for this clinical investigation?

"This medical trial seeks to enrol 12 infants, between the day of birth and one year old, who are currently diagnosed with Alagille Syndrome. Further criteria includes a body weight of no less than 2.5 kilograms, gestational age at 36 weeks or higher for those born after 32-36 weeks gestation, and an age range from 31 days up until twelve months when registering for the study (USA participants) or beginning baseline visits (international participants)."

Answered by AI

Is Maralixibat being tested under any other research protocols?

"Currently, 4 clinical trials are in progress for Maralixibat. 1 of these studies has reached Phase 3 and is being conducted primarily from Zapopan, Louisiana but also at other sites across 81 locations."

Answered by AI

How many locations are conducting this experiment?

"This research is occurring at 7 different facilities, including Los Angeles, Pittsburgh and Washington. It may be wise to register for a trial near your residence in order to curb the travel requirements associated with participation."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).

2021. "A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04729751.

All > Liver Disease