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An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

Primary Purpose

Progressive Familial Intrahepatic Cholestasis (PFIC)

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Maralixibat
Sponsored by
Mirum Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Progressive Familial Intrahepatic Cholestasis (PFIC) focused on measuring Cholestasis, Maralixibat, Mutation, PFIC, PFIC2, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Digestive System Diseases, Pediatric

Eligibility Criteria

1 Year - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Children's Hospital Los Angeles
  • Medstar Georgetown University Hospital
  • Advent Health
  • Children's Hospital at Montefiore
  • Cincinnati Children's Hospital
  • Children Hospital of Pittsburgh
  • Medical University of South Carolina
  • University of Texas Southwestern Medical Center
  • University of Texas, Health Science Center San Antonio
  • Seattle Children's Hospital
  • Hospital Italiano de Buenos Aires
  • Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
  • Cliniques Universitaires Saint-Luc
  • Sociedade Beneficente de Senhoras Hospital Sírio-Libanês
  • University of Alberta - Women and Children's Health Research Institute
  • Fundacion Cardioinfantil
  • Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon
  • CHU de Toulouse - Hôpital des Enfants
  • Medizinische Hochschule
  • Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria
  • Ospedale Pediatrico bambino Gesu'
  • Hotel Dieu de France, Alfred Naccache
  • Consultario de Joshue David Covarrubias Esquer
  • Instytut Pomnik Centrum, Zdrowia Dziecka
  • KK Women's and Children's Hospital
  • Koc University Hospital
  • Birmingham Children's Hospital
  • King's College Hospital NHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Maralixibat

Arm Description

All subjects will receive Maralixibat oral solution

Outcomes

Primary Outcome Measures

Incidence of Treatment Emergent Adverse Events (TEAEs) during the study

Secondary Outcome Measures

Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score
Maintenance of treatment effect based on the average morning ItchRO(Obs)™ severity scores over the time
Mean change from baseline over time in serum bile acid (sBA) levels
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) frequency score
Mean change from baseline over time in height and weight z-scores

Full Information

First Posted
December 2, 2019
Last Updated
October 3, 2023
Sponsor
Mirum Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04185363
Brief Title
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

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
January 8, 2020 (Actual)
Primary Completion Date
September 2024 (Anticipated)
Study Completion Date
September 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mirum Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Progressive Familial Intrahepatic Cholestasis (PFIC)
Keywords
Cholestasis, Maralixibat, Mutation, PFIC, PFIC2, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Digestive System Diseases, Pediatric

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
90 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Maralixibat
Arm Type
Experimental
Arm Description
All subjects will receive Maralixibat oral solution
Intervention Type
Drug
Intervention Name(s)
Maralixibat
Intervention Description
All subjects will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) twice daily
Primary Outcome Measure Information:
Title
Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
Time Frame
From baseline through time of interim analysis, up to 120 weeks
Secondary Outcome Measure Information:
Title
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
Title
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
Title
Mean change from baseline over time in serum bile acid (sBA) levels
Time Frame
From baseline through study completion, up to approximately 4 years
Title
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
Title
Mean change from baseline over time in height and weight z-scores
Time Frame
From baseline through study completion, up to approximately 4 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Medstar Georgetown University Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
Advent Health
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Children's Hospital at Montefiore
City
Bronx
State/Province
New York
ZIP/Postal Code
10461
Country
United States
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Children Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
University of Texas, Health Science Center San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Hospital Italiano de Buenos Aires
City
Buenos Aires
Country
Argentina
Facility Name
Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
City
Wien
Country
Austria
Facility Name
Cliniques Universitaires Saint-Luc
City
Brussels
Country
Belgium
Facility Name
Sociedade Beneficente de Senhoras Hospital Sírio-Libanês
City
São Paulo
Country
Brazil
Facility Name
University of Alberta - Women and Children's Health Research Institute
City
Edmonton
State/Province
Alberta
Country
Canada
Facility Name
Fundacion Cardioinfantil
City
Bogotá
Country
Colombia
Facility Name
Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon
City
Lyon
Country
France
Facility Name
CHU de Toulouse - Hôpital des Enfants
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
Medizinische Hochschule
City
Hanover
Country
Germany
Facility Name
Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria
City
Bergamo
ZIP/Postal Code
24127
Country
Italy
Facility Name
Ospedale Pediatrico bambino Gesu'
City
Roma
Country
Italy
Facility Name
Hotel Dieu de France, Alfred Naccache
City
Beirut
Country
Lebanon
Facility Name
Consultario de Joshue David Covarrubias Esquer
City
Zapopan
Country
Mexico
Facility Name
Instytut Pomnik Centrum, Zdrowia Dziecka
City
Warsaw
Country
Poland
Facility Name
KK Women's and Children's Hospital
City
Singapore
Country
Singapore
Facility Name
Koc University Hospital
City
Istanbul
ZIP/Postal Code
34010
Country
Turkey
Facility Name
Birmingham Children's Hospital
City
Birmingham
Country
United Kingdom
Facility Name
King's College Hospital NHS Foundation Trust
City
London
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
https://ghr.nlm.nih.gov/condition/progressive-familial-intrahepatic-cholestasis
Description
Genetics Home Reference - PFIC
URL
https://clinicaltrials.gov/ct2/info/fdalinks
Description
US FDA Resources
URL
https://mirumpharma.com/
Description
Mirum Pharmaceuticals homepage

Learn more about this trial

An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)

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