Toggle navigation
Lebanon Clinical Trials Registry
Home
About Us
FAQs
Contact Us
Search Trials
Register
Log in
User Guide
Trial details
You are here
Home
Search Trials
Trial details
Trial details
An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
Current status:
Approved
|
Date registered:
21/03/2021
Trial version(s)
Current: 03/03/2021
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2021034759
Protocol number
MRX-503
MOH registration number
NCT04185363
Trial already registered with the MoPH
Study registered at the country of origin
Study registered in clinicaltrials.gov
Type of registration
Prospective
Date of registration in national regulatory agency
04/12/2019
Primary sponsor
Mirum Pharmaceuticals Inc
Primary sponsor: Country of origin
California
Public title
An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
Acronym
Scientific title
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
Acronym
Brief summary of the study: English
This is an open-label, multicenter, Phase 3 study to evaluate the long-term safety and efficacy of maralixibat in the treatment of pediatric subjects with PFIC.
Brief summary of the study: Arabic
هذه دراسة مفتوحة ، متعددة المراكز ، المرحلة 3 لتقييم السلامة على المدى الطويل وفعالية maralixibat في علاج الأطفال الذين يعانون من PFIC.
Health conditions/problem studied: Specify
Progressive Familial Intrahepatic Cholestasis (PFIC)
Interventions: Specify
Drug: Maralixibat Dose, route, frequency: Subjects will receive maralixibat oral solution based on their individual body weight, up to 600 μg/kg BID.
Key inclusion and exclusion criteria: Inclusion criteria
1. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee. 2. Completion of study MRX-502; treatment interruption between MRX-502 and MRX-503 should be avoided. Subjects who do not complete the study MRX-503 Baseline Visit (Day 0) on the same day as the study MRX-502 EOT Visit will be considered for participation in study MRX-503 only after discussion with the Medical Monitor. 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 through 30 days after the last dose of maralixibat. 4. Females of childbearing potential must have a negative urine pregnancy test at the Baseline Visit (Day 0). 5. Access to email or telephone for scheduled remote visits. 6. Ability to read and understand the questionnaires (both caregivers and subjects above the age of assent). 7. Access to consistent caregiver(s) during the study. 8. Subject and caregiver willingness to comply with all study visits and requirements.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
1
Key inclusion and exclusion criteria: Age maximum
18
Key inclusion and exclusion criteria: Exclusion criteria
1.Any female who is pregnant or lactating or who is planning to become pregnant. 2. Administration of prohibited medication between the MRX-502 EOT visit and the MRX-503 Baseline Visit (Day 0). 3. History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability, or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment. 4. Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat. 5. Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study. 6. Cognitive impairment of the subject or caregiver that would, in the opinion of the Investigator, preclude appropriate understanding of study information and compliance with study procedures.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Other
Trial scope: Specify scope
Long Term safety and Efficacy
Study design: Allocation
N/A
Study design: Masking
Open (masking not used)
Study design: Control
N/A
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
No
Name of IMP
Maralixbat
Type of IMP
Others
Type of IMP: Specify
Pharmaceutical – Chemical
Pharmaceutical class
Maralixibat is an inhibitor of the apical sodium-dependent bile acid transporter/ileal bile acid transporter/solute carrier family 10 (sodium/bile acid cotransporter family) member 2 (ASBT/IBAT/SLC10A2), a transmembrane protein localized on the luminal surface of ileal enterocytes.
Therapeutic indication
Progressive familial intrahepatic cholestasis (PFIC)
Therapeutic benefit
The overall safety, tolerability, and preliminary efficacy of maralixibat in ongoing and completed studies indicate that there is a positive benefit-to-risk profile for the treatment of rare pediatric cholestatic liver diseases. Given the clinical outcomes associated with PFIC, including the negative impact on patients’ and caregivers’ quality of life, and the fact that there are currently no approved treatments, there is a high unmet medical need for a novel treatment for this disease.
Biospecimen retention
None retained
Biospecimen description
Blood samples
Target sample size
90
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
01/05/2021
Date of study closure: Type
Anticipated
Date of study closure: Date
30/12/2022
Recruitment status
Other
Recruitment status: Specify
Enrolling by invitation
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
The Sponsor and/or its representatives accessing the records and data will take all reasonable precautions in accordance with applicable laws, regulations, and guidelines to maintain the confidentiality of subjects’ identities. Subjects are assigned a unique identifying number; however, their initials and date of birth may also be collected, if permitted under local laws governing privacy
Additional data URL
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
US NCT
NCT04185363
Sources of Monetary or Material Support
Name
Mirum Pharmaceuticals Inc. 950 Tower LaneFoster City, CA 94404
Secondary Sponsors
Name
N/A
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Hanen
Hamid
City: Aley, Town: Bchamoun, Street: Yanar Street, Building 33, Ground Floor
Lebanon
+96181021910
Hanen.hamid@clinart.net
Clinart
Scientific
Adib
Moukarzel
HDF
Lebanon
009613516060
adib.moukarzel@usj.edu.lb
Hotel Dieu du France
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Hotel Dieu De France
Adib Moukarzel
Gastroenterology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
30/03/2020
Nancy Choucair Alam
nancy.alam@usj.edu.lb
961 1 421 000 ext 2335
Countries of Recruitment
Name
Lebanon
Argentina
Colombia
United Kingdom
United States of America
Austria
Belgium
Brazil
Canada
France
Germany
Italy
Mexico
Poland
Singapore
Turkey
Hungary
Health Conditions or Problems Studied
Condition
Code
Keyword
Progressive Familial Intrahepatic Cholestasis
2-Propanol (T51.2)
(PFIC)
Interventions
Intervention
Description
Keyword
Maralixibat Chloride
Inhibitor of the apical sodium-dependent bile acid transporter/ileal bile acid transporter/solute carrier family 10 (sodium/bile acid cotransporter family) member 2 (ASBT/IBAT/SLC10A2)
Maralixibat
Primary Outcomes
Name
Time points
Measure
Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
changes from non-serious to serious
Severity of AE
Key Secondary Outcomes
Name
Time points
Measure
Mean change from baseline over time in serum bile acid
Normalisation of sBA
sBA levels
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial