Back to results

MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study (MERGE)

Primary Purpose

Cholestatic Liver Disease

Status

Active

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Maralixibat

About this trial

This is an interventional treatment trial for Cholestatic Liver Disease focused on measuring PFIC, ALGS, Maralixibat, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Digestive System Diseases, Pediatric

Eligibility Criteria

12 Months - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria Subjects will need to meet all criteria below to be considered eligible for the study.

Provide informed consent and assent (as applicable) per the Institutional Review Board/Ethics Committee (IRB/EC).
Previously participated in a maralixibat study and with approval of the Medical Monitor. Previous participation is defined as:
- Having completed the EOT Visit, for subjects coming from the maralixibat Phase 2 studies.
- Having completed the entire duration of the study (i.e., core and extension, if applicable), for subjects coming from the maralixibat Phase 3 studies.
At least 1 year of age
Males, and females of non-childbearing potential. Males and non-pregnant, non-lactating females of childbearing potential who are sexually active must agree to use acceptable contraception during the study and 30 days following the last dose of the study medication. Females of childbearing potential must have a negative pregnancy test.
Caregivers (and/or age appropriate subjects) must have access to email or phone for scheduled remote visits if applicable.
Subject and caregiver willingness to comply with all study visits and requirements.

Exclusion Criteria

A subject will be excluded from the study if any of the following exclusion criteria are met:

Experienced an AE or SAE related to maralixibat during the lead-in protocol that led to permanent discontinuation of the subject from maralixibat.
Any conditions or abnormalities (including laboratory abnormalities) which, in the opinion of the Investigator or Medical Monitor may compromise the safety of the subject or interfere with the subject participating in or completing the study.
History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator or Sponsor medical monitor, could compromise the validity of informed consent, compromise the safety of the subject, or lead to non-adherence with the study protocol or inability to conduct the study procedures.

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Maralixibat

Arm Description

Participants will all receive Maralixibat oral solution

Outcomes

Primary Outcome Measures

Frequency of reported adverse events AEs

Treatment-emergent AEs (TEAEs) will be defined as AEs that start or deteriorate on or after the first dose of study medication and no later than 14 days following the last dose of study medication or reported through the End of Trial ( EOT/ET) Visit. In this study, all AEs will be considered TEAEs.

Secondary Outcome Measures

Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ severity score (ALGS and PFIC)

Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ frequency score (ALGS and PFIC)

Evaluate the long-term effect of maralixibat on pruritus severity using in the Clinician Scratch Scale (CSS) as change from baseline over the course of the study

Evaluate the long-term effect of maralixibat on serum bile acid levels

Evaluate the long-term effect of maralixibat on time to liver-associated outcomes reported as a mean change from baseline (i.e., partial external biliary diversion [PEBD] or liver transplantation)

Evaluate the long-term effects of maralixibat on growth

Change from baseline in height and weight, summarized as a Z-score over the course of the study

Change from maralixibat baseline over the course of the study in mean total serum bilirubin

Full Information

NCT ID

NCT04168385

First Posted

October 1, 2019

Last Updated

October 3, 2023

Sponsor

Mirum Pharmaceuticals, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04168385

Brief Title

MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study

Acronym

MERGE

Official Title

MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

January 16, 2020 (Actual)

Primary Completion Date

January 2024 (Anticipated)

Study Completion Date

January 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Mirum Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia.

Detailed Description

This is a multicenter, open-label study of maralixibat in subjects diagnosed with cholestatic liver disease (including, but not limited to ALGS, PFIC or Biliary Atresia) who have previously participated in a maralixibat clinical study. All subjects will receive maralixibat in this study.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cholestatic Liver Disease

Keywords

PFIC, ALGS, Maralixibat, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Digestive System Diseases, Pediatric

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

52 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Maralixibat

Arm Type

Experimental

Arm Description

Participants will all receive Maralixibat oral solution

Intervention Type

Drug

Intervention Name(s)

Maralixibat

Other Intervention Name(s)

Formerly LUM001 and SHP625

Intervention Description

Maralixibat chloride oral solution orally twice daily (up to 1200* mcg/kg/day), and according to indication. *equivalent to 1140 mcg/kg/day maralixibat

Primary Outcome Measure Information:

Title

Frequency of reported adverse events AEs

Description

Time Frame