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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04168385
Ethics application status
Date submitted
1/10/2019
Date registered
19/11/2019

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

\*equivalent to 1140 mcg/kg/day maralixibat
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency of reported adverse events AEs
Timepoint [1] 0 0
From baseline through study completion, up to approximately 3 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 (ALGS and PFIC)
Timepoint [1] 0 0
From baseline through study completion, up to approximately 3 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 (ALGS and PFIC)
Timepoint [2] 0 0
From baseline through study completion, up to approximately 3 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 approximately 3 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 approximately 3 years
Secondary outcome [5] 0 0
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)
Timepoint [5] 0 0
From baseline to occurrence of liver associated event, up to approximately 3 years
Secondary outcome [6] 0 0
Evaluate the long-term effects of maralixibat on growth
Timepoint [6] 0 0
Through study completion, up to approximately 3 years
Secondary outcome [7] 0 0
Change from maralixibat baseline over the course of the study in mean total serum bilirubin
Timepoint [7] 0 0
Change from baseline through study completion, up to approximately 3 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. At least 1 year of age
4. 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.
5. Caregivers (and/or age appropriate subjects) must have access to email or phone for scheduled remote visits if applicable.
6. Subject and caregiver willingness to comply with all study visits and requirements.
Minimum age
12 Months
Maximum age
No limit
Sex
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
NA
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/01/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2024
Actual
Sample size
Target
Accrual to date
Final
52
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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04168385