An Extension Study of Maralixibat in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC)
- Conditions
- Progressive Familial Intrahepatic Cholestasis (PFIC)
- Interventions
- Registration Number
- NCT04185363
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Brief Summary
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
- Detailed Description
The study will be conducted at multiple sites in North America, Europe, Asia, and South America.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 90
- Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
- Completion of study MRX-502
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Maralixibat Maralixibat All subjects will receive Maralixibat oral solution
- Primary Outcome Measures
Name Time Method Incidence of Treatment Emergent Adverse Events (TEAEs) during the study From baseline through time of interim analysis, up to 120 weeks
- Secondary Outcome Measures
Name Time Method Maintenance of treatment effect based on the average morning ItchRO(Obs)™ severity scores over the time From baseline through study completion, up to approximately 4 years Mean change from baseline over time in serum bile acid (sBA) levels From baseline through study completion, up to approximately 4 years Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score From baseline through study completion, up to approximately 4 years Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score From baseline through study completion, up to approximately 4 years Mean change from baseline over time in height and weight z-scores From baseline through study completion, up to approximately 4 years
Trial Locations
- Locations (28)
KK Women's and Children's Hospital
🇸🇬Singapore, Singapore
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Medstar Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States
Advent Health
🇺🇸Orlando, Florida, United States
Children's Hospital at Montefiore
🇺🇸Bronx, New York, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Children Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
University of Texas Southwestern Medical Center
🇺🇸Dallas, Texas, United States
University of Texas, Health Science Center San Antonio
🇺🇸San Antonio, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Hospital Italiano de Buenos Aires
🇦🇷Buenos Aires, Argentina
Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
🇦🇹Wien, Austria
Cliniques Universitaires Saint-Luc
🇧🇪Brussels, Belgium
Sociedade Beneficente de Senhoras Hospital Sírio-Libanês
🇧🇷São Paulo, Brazil
University of Alberta - Women and Children's Health Research Institute
🇨🇦Edmonton, Alberta, Canada
Fundacion Cardioinfantil
🇨🇴Bogotá, Colombia
Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon
🇫🇷Lyon, France
CHU de Toulouse - Hôpital des Enfants
🇫🇷Toulouse, France
Medizinische Hochschule
🇩🇪Hanover, Germany
Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria
🇮🇹Bergamo, Italy
Ospedale Pediatrico bambino Gesu'
🇮🇹Roma, Italy
Hotel Dieu de France, Alfred Naccache
🇱🇧Beirut, Lebanon
Consultario de Joshue David Covarrubias Esquer
🇲🇽Zapopan, Mexico
Instytut Pomnik Centrum, Zdrowia Dziecka
🇵🇱Warsaw, Poland
Koc University Hospital
🇹🇷Istanbul, Turkey
Birmingham Children's Hospital
🇬🇧Birmingham, United Kingdom
King's College Hospital NHS Foundation Trust
🇬🇧London, United Kingdom