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IBAT Inhibitor

Maralixibat for Cholestasis

Phase 3
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
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline through study completion, up to approximately 4 years
Awards & highlights

Study Summary

This trial will study the long-term safety and tolerability of the drug maralixibat in people with no medical education.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While specific side effects are not listed here, participants will be monitored for any adverse reactions since this study aims to assess the long-term safety and tolerability of Maralixibat.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline through study completion, up to approximately 4 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline through study completion, up to approximately 4 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
Secondary outcome measures
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score
Maintenance of treatment effect based on the average morning ItchRO(Obs)™ severity scores over the time
+2 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 deficiency
9%
Vitamin D decreased
6%
Vitamin E deficiency
6%
Gastroenteritis
6%
Coronavirus infection
6%
Upper respiratory tract 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

Trial Design

1Treatment groups
Experimental Treatment
Group I: MaralixibatExperimental Treatment1 Intervention
All subjects will receive Maralixibat oral solution
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
1,556 Total Patients Enrolled

Media Library

Maralixibat (IBAT Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04185363 — Phase 3
Progressive Familial Intrahepatic Cholestasis Research Study Groups: Maralixibat
Progressive Familial Intrahepatic Cholestasis Clinical Trial 2023: Maralixibat Highlights & Side Effects. Trial Name: NCT04185363 — Phase 3
Maralixibat (IBAT Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04185363 — Phase 3

Frequently Asked Questions

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

What other investigational therapies has Maralixibat been tested in?

"Maralixibat is being trialled in 4 different studies, one of which is in Phase 3. The majority of locations for these studies are in Zapopan, but there are also active trials in 81 other locations."

Answered by AI

What are the possible adverse effects of taking Maralixibat?

"Maralixibat is considered to be a safe medication because it has undergone Phase 3 clinical trials."

Answered by AI

Does this experiment include elderly individuals as test subjects?

"According to the rules established for this clinical trial, the youngest an eligible patient can be is 1 year old while the maximum age is 18."

Answered by AI

Are recruitment efforts for this trial still underway?

"According to the latest information available on clinicaltrials.gov, this particular trial is not looking for new patients at this time. The study was first posted on 2020-01-08 and was last edited on 2022-08-15. However, there are 29 other trials that are currently open for recruitment."

Answered by AI

How many men and women are enrolled in this clinical trial currently?

"This clinical trial is not recruiting patients at the moment. The trial was originally posted on 2020-01-08 and was most recently edited on 2022-08-15. However, there are other studies you may be eligible for depending on your condition. For example, there are 25 clinical trials actively recruiting participants with progressive familial intrahepatic cholestasis (pfic) and 4 studies for Maralixibat actively searching for participants."

Answered by AI

Has this type of clinical trial been conducted before?

"Maralixibat has been under investigation since 2019. The first clinical study, sponsored by Mirum Pharmaceuticals, occurred that year and included 52 participants. After the positive results of the initial trial, Maralixibat received Phase 2 drug approval. Currently, there are 4 live trials involving Maralixibat taking place in 26 cities and 20 countries."

Answered by AI

How do I sign up for this research project?

"Eligible participants for this clinical trial must have progressive familial intrahepatic cholestasis (pfic) and be between 1 and 18 years old. Up to 90 patients will be enrolled in this study."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
2020. "An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04185363.
~7 spots leftby Sep 2024
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