Clinical trial NCT04185363: An Extension Study of Maralixibat in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC)
Official 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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC)
Keywords: Cholestasis
Maralixibat
Mutation
PFIC
PFIC2
Bile Duct Diseases
Liver Diseases
Biliary Tract Diseases
Digestive System Diseases
Pediatric
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-12-04
Enrollment: 90
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: Key Inclusion Criteria:

1. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
2. Completion of study MRX-502

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

Primary outcomes:
measure: Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
timeFrame: From baseline through time of interim analysis, up to 120 weeks
Secondary outcomes:
measure: Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score
timeFrame: From baseline through study completion, up to approximately 4 years
measure: Maintenance of treatment effect based on the average morning ItchRO(Obs)™ severity scores over the time
timeFrame: From baseline through study completion, up to approximately 4 years
measure: Mean change from baseline over time in serum bile acid (sBA) levels
timeFrame: From baseline through study completion, up to approximately 4 years
measure: Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score
timeFrame: From baseline through study completion, up to approximately 4 years
measure: Mean change from baseline over time in height and weight z-scores
timeFrame: From baseline through study completion, up to approximately 4 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Washington
country: United States
facility: Medstar Georgetown University Hospital
state: District of Columbia
city: Orlando
country: United States
facility: Advent Health
state: Florida
city: Bronx
country: United States
facility: Children's Hospital at Montefiore
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Pittsburgh
country: United States
facility: Children Hospital of Pittsburgh
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: San Antonio
country: United States
facility: University of Texas, Health Science Center San Antonio
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Buenos Aires
country: Argentina
facility: Hospital Italiano de Buenos Aires
city: Wien
country: Austria
facility: Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: São Paulo
country: Brazil
facility: Sociedade Beneficente de Senhoras Hospital Sírio-Libanês
city: Edmonton
country: Canada
facility: University of Alberta - Women and Children's Health Research Institute
state: Alberta
city: Bogotá
country: Colombia
facility: Fundacion Cardioinfantil
city: Lyon
country: France
facility: Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon
city: Toulouse
country: France
facility: CHU de Toulouse - Hôpital des Enfants
city: Hanover
country: Germany
facility: Medizinische Hochschule
city: Bergamo
country: Italy
facility: Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria
city: Roma
country: Italy
facility: Ospedale Pediatrico bambino Gesu'
city: Beirut
country: Lebanon
facility: Hotel Dieu de France, Alfred Naccache
city: Zapopan
country: Mexico
facility: Consultario de Joshue David Covarrubias Esquer
city: Warsaw
country: Poland
facility: Instytut Pomnik Centrum, Zdrowia Dziecka
city: Singapore
country: Singapore
facility: KK Women&#39;s and Children&#39;s Hospital
city: Istanbul
country: Turkey
facility: Koc University Hospital
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: London
country: United Kingdom
facility: King's College Hospital NHS Foundation Trust
Study officials:
References: