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Ion Transporter Inhibitor

Maralixibat for Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC Trial)

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 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up mmrm model was used with inputs of the average changes from baseline (bl) to weeks 1-6, 7-10, 11-14, 15-18, 19-22, 23-26, and other covariates. the average change from bl over weeks 15-26 is calculated in the model and presented as a single number.

Awards & highlights

MARCH-PFIC Trial Summary

This study is evaluating whether a medication may help treat a rare liver disease.

Eligible Conditions

Progressive Familial Intrahepatic Cholestasis

MARCH-PFIC Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ mmrm model was used with inputs of the average changes from baseline (bl) to weeks 1-6, 7-10, 11-14, 15-18, 19-22, 23-26, and other covariates. the average change from bl over weeks 15-26 is calculated in the model and presented as a single number.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~mmrm model was used with inputs of the average changes from baseline (bl) to weeks 1-6, 7-10, 11-14, 15-18, 19-22, 23-26, and other covariates. the average change from bl over weeks 15-26 is calculated in the model and presented as a single number.

This trial's timeline: 3 weeks for screening, Varies for treatment, and mmrm model was used with inputs of the average changes from baseline (bl) to weeks 1-6, 7-10, 11-14, 15-18, 19-22, 23-26, and other covariates. the average change from bl over weeks 15-26 is calculated in the model and presented as a single number. 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 the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort

Secondary outcome measures

Mean Change in Total sBA Level in Participants With PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)

Mean Change in Total sBA Level in the Primary Cohort.

Mean Change in the Average Morning ItchRO(Obs) Severity Score in Participants With PFIC (PFIC1, Nt-PFIC2, PFIC3, PFIC4 and PFIC6)

+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

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

MARCH-PFIC Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MaralixibatExperimental Treatment1 Intervention

Participants will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) orally twice daily for 26 weeks.

Group II: PlaceboPlacebo Group1 Intervention

Participants will receive placebo matched to maralixibat oral solution twice daily for 26 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Maralixibat

2015

Completed Phase 3

~260

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Mirum Pharmaceuticals, Inc.Lead Sponsor

29 Previous Clinical Trials

1,553 Total Patients Enrolled

Frequently Asked Questions

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

Recent research and studies

clinicaltrials.govStudy

A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)

2019. "A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03905330.

~16 spots leftby Apr 2025

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