Main
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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:
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EUCTR |
Last refreshed on:
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7 October 2024 |
Main ID: |
EUCTR2019-002755-42-PL |
Date of registration:
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08/01/2020 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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MERGE: Maralixibat Extension Safety Study Providing Long-term Treatment to Subjects with Cholestatic Liver Disease.
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Scientific title:
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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:
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Target sample size:
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109 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-002755-42 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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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
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
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Countries of recruitment
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Australia
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Belgium
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Canada
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France
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Poland
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Spain
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United Kingdom
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United States
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Contacts
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Name:
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Chief Scientific Officer
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Address:
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950 Tower Lane, Suite 1050
CA 94404
Foster City California
United States |
Telephone:
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16506674085 |
Email:
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medinfo@mirumpharma.co |
Affiliation:
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Mirum Pharmaceuticals Inc. |
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Name:
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Chief Scientific Officer
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Address:
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950 Tower Lane, Suite 1050
CA 94404
Foster City California
United States |
Telephone:
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16506674085 |
Email:
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medinfo@mirumpharma.co |
Affiliation:
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Mirum Pharmaceuticals Inc. |
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Key inclusion & exclusion criteria
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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,MRX-701or other open-label maralixibat studies). •Previously early terminated from a maralixibat study for reasons other than safety, and received permission from the Medical Monitor to enroll, after completing all screening procedures and confirmation of eligibility •Having reached the stable-dose phase of maralixibat in the open-label extension of a previous study and are =1 year of age 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.
Are the trial subjects under 18? yes Number of subjects for this age range: 104 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
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Health Condition(s) or Problem(s) studied
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Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
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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) and Biliary Atresia. 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
MedDRA version: 20.0
Level: LLT
Classification code 10004653
Term: Biliary atresia
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Intervention(s)
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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-
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Primary Outcome(s)
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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 total serum bilirubin •Evaluate the long-term effect of maralixibat on time to liver-associated outcomes (i.e., partial external biliary diversion (PEBD) or liver transplantation) •Evaluate the long-term effects of maralixibat on growth
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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 •Change from maralixibat baseline in neurodevelopmental assessment (biliary atresia)
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Main Objective: Evaluate the long-term safety of maralixibat in subjects with cholestatic liver disease including, but not limited to, ALGS and PFIC and Biliary Atresia.
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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.
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Secondary Outcome(s)
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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.
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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 (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) •Change from maralixibat baseline over the course of the study in the Clinician Scratch Scale Score •Change from maralixibat baseline over the course of the study in sBA levels •Change from maralixibat baseline over the course of the study in mean total serum bilirubin •Time from maralixibat baseline to liver-associated outcomes (PEBD surgery, listing for liver transplantation, liver decompensation [hepatic encephalopathy, variceal bleeding, ascites, and spontaneous bacterial peritonitis] events, hepatocellular carcinoma, death from a liver associated event). •Comparison between treatment responders (defined as sBA normalization/= 70% change from maralixibat baseline (CFMB) 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 (ALGS and PFIC) •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 triceps skinfold, head circumference (until age 2 years), and mid arm circumference z-score over the course of the study (for subjects with biliary atresia) •Change from maralixibat baseline in xanthomas over the course of the study, as measured by the Clinician Xanthoma Scale (ALGS)
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Secondary ID(s)
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2019-002755-42-BE
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MRX-800
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Source(s) of Monetary Support
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Mirum Pharmaceuticals Inc.
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Ethics review
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Status:
Approval date:
Contact:
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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