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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 11 March 2020
Main ID:  EUCTR2019-002755-42-PL
Date of registration: 08/01/2020
Prospective Registration: Yes
Primary sponsor: Mirum Pharmaceuticals Inc.
Public title: MERGE: Maralixibat Extension Safety Study Providing Long-term Treatment to Subjects with Cholestatic Liver Disease.
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.
Date of first enrolment:
Target sample size: 53
Recruitment status: NA
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-002755-42
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no
Countries of recruitment
Australia Belgium Canada France Poland Spain United Kingdom United States
Contacts
Name: Chief Scientific Officer   
Address:  950 Tower Lane, Suite 1050 CA 94404 Foster City California United States
Telephone: 16506674085
Email: medinfo@mirumpharma.co
Affiliation:  Mirum Pharmaceuticals Inc.
Name: Chief Scientific Officer   
Address:  950 Tower Lane, Suite 1050 CA 94404 Foster City California United States
Telephone: 16506674085
Email: medinfo@mirumpharma.co
Affiliation:  Mirum Pharmaceuticals Inc.
Key inclusion & exclusion criteria
Inclusion criteria:
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 (LUM001-303, LUM001-304, and LUM001-305 in ALGS and LUM001-501 in PFIC).
•Having completed the entire duration of the study (i.e., core and extension, if applicable), for subjects coming from other maralixibat studies (e.g., MRX 503 and other open-label maralixibat 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.

Are the trial subjects under 18? yes
Number of subjects for this age range: 53
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
1.Experienced an adverse event (AE) or serious adverse event (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 nonadherence with the study protocol or inability to conduct the study procedures.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Long-term safety study with Maralixibat, in treatment of subjects with cholestatic liver disease including, but not limited to, Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).
MedDRA version: 20.0 Level: PT Classification code 10076033 Term: Progressive familial intrahepatic cholestasis System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0 Level: PT Classification code 10053870 Term: Alagille syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Intervention(s)

Product Name: Maralixibat (formely SHP625 or LUM001)
Pharmaceutical Form: Oral solution
INN or Proposed INN: MARALIXIBAT CHLORIDE
CAS Number: 228113-66-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 5-

Product Name: Maralixibat (formely SHP625 or LUM001)
Pharmaceutical Form: Oral solution
INN or Proposed INN: MARALIXIBAT CHLORIDE
CAS Number: 228113-66-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Product Name: Maralixibat (formely SHP625 or LUM001)
Pharmaceutical Form: Oral solution
INN or Proposed INN: MARALIXIBAT CHLORIDE
CAS Number: 228113-66-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 15-

Product Name: Maralixibat (formely SHP625 or LUM001)
Pharmaceutical Form: Oral solution
INN or Proposed INN: MARALIXIBAT CHLORIDE
CAS Number: 228113-66-4
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 20-

Primary Outcome(s)
Main Objective: Evaluate the long-term safety of maralixibat in subjects with cholestatic liver disease including, but not limited to, ALGS and PFIC.
Primary end point(s): The following safety and tolerability endpoints will be assessed:
•AEs including serious, non-serious, related, non-related AEs
•Clinical laboratory tests (hematology, chemistry, urinalysis; serum pregnancy test, if appropriate)
•Vital signs (temperature, systolic and diastolic blood pressure, heart rate, respiratory rate, weight and height assessments)
•Physical examination
•Concomitant treatment/medication usage
Secondary Objective: •Evaluate the long-term effect of maralixibat on pruritus
•Evaluate the long-term effect of maralixibat on serum bile acid levels
•Evaluate the long-term effect of maralixibat on time to liver-associated events (i.e., partial external biliary diversion (PEBD) or liver transplantation)
•Evaluate the long-term effects of maralixibat on growth
Timepoint(s) of evaluation of this end point: A Data Monitoring Committee (DMC) will review safety and study data at specified intervals for the duration of the study.
Secondary Outcome(s)
Secondary end point(s): The efficacy endpoints:
•Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ severity score
•Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ frequency score
•Change from maralixibat baseline over the course of the study in the Clinician Scratch Score
•Change from maralixibat baseline over the course of the study in sBA levels
•Time from maralixibat baseline to liver-associated events (PEBD surgery, listing for liver transplantation, liver decompensation [hepatic encephalopathy, variceal bleeding, ascites, and spontaneous bacterial peritonitis] events, HCC, death from a liver associated event).
•Comparison between treatment responders (defined as sBA normalization/= 70% CFMB (change from maralixibat baseline) AND = 1.0 CFMB ItchRO(Obs)™) and partial/non-responder (PFIC)
•Comparison between treatment responders (defined as sBA = 50% CFMB (change from maralixibat baseline) AND =1.0 CFMB ItchRO(Obs)™) and partial/non-responder (ALGS)
•Proportion of days at or below 1.0 point on the weekly morning ItchRO(Obs)™ severity score
•Durability of treatment response
•Change from maralixibat baseline in height and weight Z-score over the course of the study
•Change from maralixibat baseline in xanthomas over the course of the study, as measured by the Clinician Xanthoma Scale (ALGS)
Timepoint(s) of evaluation of this end point: All such analyses will be interpreted cautiously and not used for formal inference, although inferential statistics may be used as part of the data summary.
Secondary ID(s)
2019-002755-42-GB
MRX-800
Source(s) of Monetary Support
Mirum Pharmaceuticals Inc.
Secondary Sponsor(s)
Ethics review
Status:
Approval date:
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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