COVID-19 studies are our top priority. For all other trials, there is a 4-week delay in processing a trial submitted/resubmitted to the ANZCTR and additional delays for updates of registered trials. We appreciate your patience.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04168385




Registration number
NCT04168385
Ethics application status
Date submitted
1/10/2019
Date registered
19/11/2019
Date last updated
9/06/2020

Titles & IDs
Public 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
Scientific 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
Secondary ID [1] 0 0
MRX-800
Universal Trial Number (UTN)
Trial acronym
MERGE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cholestatic Liver Disease 0 0
Condition category
Condition code
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Cancer 0 0 0 0
Liver

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Maralixibat

Experimental: Maralixibat - Participants will all receive Maralixibat oral solution


Treatment: Drugs: Maralixibat
Maralixibat oral solution (up to 900 mcg/kg) orally twice daily

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
From baseline through study completion, up to 2 years
Secondary outcome [1] 0 0
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ severity score
Timepoint [1] 0 0
From baseline through study completion,up to 2 years
Secondary outcome [2] 0 0
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ frequency score
Timepoint [2] 0 0
From baseline through study completion,up to 2 years
Secondary outcome [3] 0 0
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 .
Timepoint [3] 0 0
From baseline through study completion,up to 2 years
Secondary outcome [4] 0 0
Evaluate the long-term effect of maralixibat on serum bile acid levels
Timepoint [4] 0 0
From baseline through study completion,up to 2 years
Secondary outcome [5] 0 0
Evaluate the long-term effect of maralixibat on time to liver-associated events reported as a mean change from baseline (i.e., partial external biliary diversion [PEBD] or liver transplantation)
Timepoint [5] 0 0
From baseline to occurrence of liver associated event, up to 2 years
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
Through study completion, up to 2 years

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

1. Provide informed consent and assent (as applicable) per the Institutional Review
Board/Ethics Committee (IRB/EC).

2. 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.

3. 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.

4. Caregivers (and/or age appropriate subjects) must have access to email or phone for
scheduled remote visits if applicable.

5. Subject and caregiver willingness to comply with all study visits and requirements.
Minimum age
12 Months
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

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

1. Experienced an AE or SAE related to maralixibat during the lead-in protocol that led
to permanent discontinuation of the subject from maralixibat.

2. 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.

3. 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Children's Hospital Westmead - Westmead
Recruitment hospital [2] 0 0
The Royal Children'S Hospital Melbourne - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Indiana
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania
Country [4] 0 0
United States of America
State/province [4] 0 0
Texas
Country [5] 0 0
United States of America
State/province [5] 0 0
Washington
Country [6] 0 0
Belgium
State/province [6] 0 0
Brussels
Country [7] 0 0
Canada
State/province [7] 0 0
Toronto
Country [8] 0 0
France
State/province [8] 0 0
Paris
Country [9] 0 0
Poland
State/province [9] 0 0
Warsaw
Country [10] 0 0
Spain
State/province [10] 0 0
Madrid
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Birmingham
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Leeds
Country [13] 0 0
United Kingdom
State/province [13] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Mirum Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease
including, but not limited to, Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic
Cholestasis (PFIC)
Trial website
https://clinicaltrials.gov/show/NCT04168385
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04168385