- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04185363
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
October 3, 2023 updated by: Mirum Pharmaceuticals, Inc.
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)
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
Study Overview
Status
Active, not recruiting
Intervention / Treatment
Detailed Description
The study will be conducted at multiple sites in North America, Europe, Asia, and South America.
Study Type
Interventional
Enrollment (Actual)
90
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Buenos Aires, Argentina
- Hospital Italiano de Buenos Aires
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Wien, Austria
- Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
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Brussels, Belgium
- Cliniques Universitaires Saint-Luc
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São Paulo, Brazil
- Sociedade Beneficente de Senhoras Hospital Sirio-Libanes
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Alberta
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Edmonton, Alberta, Canada
- University of Alberta - Women and Children's Health Research Institute
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Bogotá, Colombia
- Fundación Cardioinfantil
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Lyon, France
- Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon
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Toulouse, France, 31059
- CHU de TOULOUSE - Hôpital des Enfants
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Hanover, Germany
- Medizinische Hochschule
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Bergamo, Italy, 24127
- Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria
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Roma, Italy
- Ospedale Pediatrico bambino Gesu'
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Beirut, Lebanon
- Hotel Dieu de France, Alfred Naccache
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Zapopan, Mexico
- Consultario de Joshue David Covarrubias Esquer
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Warsaw, Poland
- Instytut Pomnik Centrum, Zdrowia Dziecka
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Singapore, Singapore
- KK Women's and Children's Hospital
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Istanbul, Turkey, 34010
- Koc University Hospital
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Birmingham, United Kingdom
- Birmingham Children's Hospital
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London, United Kingdom
- King's College Hospital NHS Foundation Trust
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California
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Los Angeles, California, United States, 90027
- Children's Hospital Los Angeles
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District of Columbia
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Washington, District of Columbia, United States, 20007
- MedStar Georgetown University Hospital
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Florida
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Orlando, Florida, United States, 32803
- Advent Health
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New York
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Bronx, New York, United States, 10461
- Children's Hospital at Montefiore
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital
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Pennsylvania
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Pittsburgh, Pennsylvania, United States, 15224
- Children Hospital of Pittsburgh
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South Carolina
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Charleston, South Carolina, United States, 29425
- Medical University of South Carolina
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Texas
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Dallas, Texas, United States, 75390
- University of Texas Southwestern Medical Center
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San Antonio, Texas, United States, 78229
- University of Texas, Health Science Center San Antonio
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
1 year to 18 years (Child, Adult)
Accepts Healthy Volunteers
No
Description
Key Inclusion Criteria:
- Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
- Completion of study MRX-502
Exclusion Criteria:
- Any female who is pregnant or lactating or who is planning to become pregnant
- Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0)
- 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
- 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
- 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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Maralixibat
All subjects will receive Maralixibat oral solution
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All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) twice daily
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
Time Frame: From baseline through time of interim analysis, up to 120 weeks
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From baseline through time of interim analysis, up to 120 weeks
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score
Time Frame: From baseline through study completion, up to approximately 4 years
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From baseline through study completion, up to approximately 4 years
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Maintenance of treatment effect based on the average morning ItchRO(Obs)™ severity scores over the time
Time Frame: From baseline through study completion, up to approximately 4 years
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From baseline through study completion, up to approximately 4 years
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Mean change from baseline over time in serum bile acid (sBA) levels
Time Frame: From baseline through study completion, up to approximately 4 years
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From baseline through study completion, up to approximately 4 years
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Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score
Time Frame: From baseline through study completion, up to approximately 4 years
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From baseline through study completion, up to approximately 4 years
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Mean change from baseline over time in height and weight z-scores
Time Frame: From baseline through study completion, up to approximately 4 years
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From baseline through study completion, up to approximately 4 years
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
January 8, 2020
Primary Completion (Estimated)
September 1, 2024
Study Completion (Estimated)
September 1, 2024
Study Registration Dates
First Submitted
December 2, 2019
First Submitted That Met QC Criteria
December 2, 2019
First Posted (Actual)
December 4, 2019
Study Record Updates
Last Update Posted (Actual)
October 5, 2023
Last Update Submitted That Met QC Criteria
October 3, 2023
Last Verified
October 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- MRX-503
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Progressive Familial Intrahepatic Cholestasis (PFIC)
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Mirum Pharmaceuticals, Inc.CompletedProgressive Familial Intrahepatic Cholestasis (PFIC)Italy, United States, Argentina, France, Singapore, United Kingdom, Austria, Brazil, Mexico, Lebanon, Germany, Turkey, Poland, Belgium, Canada, Colombia, Hungary
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TakedaActive, not recruitingProgressive Familial Intrahepatic Cholestasis (PFIC)Japan
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Mirum Pharmaceuticals, Inc.WithdrawnProgressive Familial Intrahepatic Cholestasis (PFIC)
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Mirum Pharmaceuticals, Inc.CompletedProgressive Familial Intrahepatic Cholestasis (PFIC)United States, United Kingdom, Poland, France
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Institute of Liver and Biliary Sciences, IndiaKEM Hospital Research Centre; Apollo Hospital, New Delhi, India; Jaslok Hospital... and other collaboratorsRecruitingProgressive Familial Intrahepatic CholestasisIndia
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University Medical Center GroningenEnrolling by invitationProgressive Familial Intrahepatic CholestasisNetherlands
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AlbireoActive, not recruitingProgressive Familial Intrahepatic CholestasisUnited States, Spain, Netherlands, United Kingdom, Turkey, Canada, Italy, Germany, France, Israel, Australia, Belgium, Poland, Saudi Arabia, Sweden
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AlbireoApproved for marketingProgressive Familial Intrahepatic CholestasisUnited States
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National Liver Institute, EgyptUnknownProgressive Familial Intrahepatic CholestasisEgypt
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Mirum Pharmaceuticals, Inc.CompletedAlagille Syndrome | Progressive Familial Intrahepatic CholestasisUnited States, Canada
Clinical Trials on Maralixibat
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TakedaActive, not recruiting
-
TakedaActive, not recruitingProgressive Familial Intrahepatic Cholestasis (PFIC)Japan
-
Mirum Pharmaceuticals, Inc.RecruitingAlagille SyndromeUnited States
-
Mirum Pharmaceuticals, Inc.CompletedProgressive Familial Intrahepatic Cholestasis (PFIC)United States, United Kingdom, Poland, France
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Mirum Pharmaceuticals, Inc.Clinigen, Inc.Approved for marketingAlagille SyndromeUnited States
-
Mirum Pharmaceuticals, Inc.CompletedAlagille SyndromeFrance, Belgium, Poland, Australia, Spain, United Kingdom
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Mirum Pharmaceuticals, Inc.Completed
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Mirum Pharmaceuticals, Inc.Lumena Pharmaceuticals, Inc.; Childhood Liver Disease Research and Education...Completed
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Mirum Pharmaceuticals, Inc.WithdrawnProgressive Familial Intrahepatic Cholestasis (PFIC)
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Mirum Pharmaceuticals, Inc.Active, not recruitingCholestatic Liver Disease | Alagille Syndrome | Progressive Familial Intrahepatic CholestasisUnited States, United Kingdom, France, Belgium, Poland, Brazil, Mexico