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IBAT Inhibitor

Maralixibat for Biliary Atresia (EMBARK 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
Clinical diagnosis of biliary atresia
Male or female subjects with body weight ≥2500 g, who are ≥21 days old and <90 days old at the time of HPE (Kasai)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline to week 26
Awards & highlights

EMBARK Trial Summary

This trial will test if maralixibat is a safe and effective treatment for infants with biliary atresia after the Kasai procedure.

Who is the study for?
This trial is for infants weighing at least 2500 grams, aged between 21 and 90 days who have undergone the Kasai procedure within the past three weeks to treat Biliary Atresia. Infants with chronic diarrhea, intolerance to feeding, other bile duct issues like sclerosing cholangitis or liver failure are excluded.Check my eligibility
What is being tested?
The study tests Maralixibat's effectiveness and safety in babies with Biliary Atresia post-Kasai surgery. It compares Maralixibat against a placebo to see if it improves outcomes after this specific liver operation.See study design
What are the potential side effects?
While not specified here, potential side effects of Maralixibat may include digestive issues since it targets bile acid processes; however, exact side effects will be monitored throughout the trial.

EMBARK Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with biliary atresia.
Select...
My baby weighs at least 2500g, is older than 21 days but younger than 90 days at the time of their Kasai procedure.
Select...
I had liver surgery or a Kasai procedure within the last 3 weeks.

EMBARK Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline to week 26
This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to week 26 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Mean change in total serum bilirubin levels
Secondary outcome measures
Mean change in total serum bile acids
Proportion of participants observed to develop clinically evident portal hypertension defined as splenomegaly and thrombocytopenia (platelet count <150 x 109/L) or clinically evident ascites or endoscopic evidence of esophageal or gastric varices.
Proportion of participants observed to have a liver-related clinical event, including liver transplantation, liver decompensation, discontinuations due to liver related events, or death.
+4 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
9%
Vitamin E decreased
9%
Constipation
9%
Vitamin D decreased
9%
Vitamin D deficiency
6%
Vitamin E deficiency
6%
Gastroenteritis
6%
Upper respiratory tract infection
6%
Coronavirus infection
6%
Vomiting
4%
Urinary tract infection
4%
Abdominal pain upper
2%
International normalised ratio increased
2%
Cholestasis
2%
Idiopathic pneumonia syndrome
100%
80%
60%
40%
20%
0%
Study treatment Arm
Maralixibat
Placebo

EMBARK Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: MaralixibatExperimental Treatment1 Intervention
Maralixibat chloride oral solution administered twice daily, up to 600* microgram per kilogram, for 26 weeks and in the OLE for all patients. *equivalent to 570 mcg/kg/day maralixibat free base
Group II: PlaceboPlacebo Group1 Intervention
Placebo oral solution for 26 weeks. All placebo participants who complete Week 26 and continue in the open label extension (OLE) will receive maralixibat after Week 26.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Maralixibat
2015
Completed Phase 3
~260

Find a Location

Who is running the clinical trial?

Mirum Pharmaceuticals, Inc.Lead Sponsor
29 Previous Clinical Trials
1,571 Total Patients Enrolled

Media Library

Maralixibat (IBAT Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04524390 — Phase 2
Biliary Atresia Research Study Groups: Placebo, Maralixibat
Biliary Atresia Clinical Trial 2023: Maralixibat Highlights & Side Effects. Trial Name: NCT04524390 — Phase 2
Maralixibat (IBAT Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04524390 — Phase 2

Frequently Asked Questions

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

To what extent has this trial been disseminated across the country?

"This clinical trial is registering participants at Le Bonheur Children's Hospital in Memphis, The University of Chicago Medical Center in Illinois, and the Children's Hospital of Philadelphia in Pennsylvania amongst 8 other research centres."

Answered by AI

Is the recruitment phase of this clinical trial still open?

"Affirmative, per the information located on clinicaltrials.gov this study is currently recruiting. This trial was initially shared on July 8th 2021 and most recently updated September 22nd 2022. 72 test subjects are being sought out from 8 distinct locations."

Answered by AI

Could I potentially qualify to take part in this clinical exploration?

"Candidates interested in this clinical trial must suffer from biliary atresia, and their age needs to range between 21 days old and 111 days. 72 participants are needed for the study's completion."

Answered by AI

Is this a pioneering research endeavor?

"At present, there are 4 active trials for Maralixibat located across 20 nations in 26 cities. The initial investigation into the drug was conducted by Mirum Pharmaceuticals Inc., concluding its Phase 2 trial with 52 participants back in 2019; since then, 18330 additional studies have been completed."

Answered by AI

How many participants are currently taking part in this medical research?

"The research team at Mirum Pharmaceuticals, Inc. requires 72 suitable participants to conduct the clinical trial. Prospective patients can apply for inclusion from Le Bonheur Children's Hospital in Memphis and The University of Chicago Medical Center in Illinois."

Answered by AI

Is the age restriction of this trial greater than two decades?

"This trial necessitates that patients who wish to enroll must be between 21 and 111 days old, in accordance with the set eligibility criteria."

Answered by AI

Could you please describe any other trials which have used Maralixibat?

"Maralixibat was first studied by Hopital Necker-Enfants Malades in 2019 and since then 18330 clinical trials have been completed. Currently, 4 additional studies are underway with the majority of these being conducted from Memphis, Tennessee."

Answered by AI

Has Maralixibat been given regulatory approval by the FDA?

"Given the Phase 2 status of Maralixibat's clinical trial, Power puts its safety rating at a conservative 2 since there is some data backing its security but none vouching for efficacy."

Answered by AI
~20 spots leftby Mar 2025