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)
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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Incidence of Treatment Emergent Adverse Events (TEAEs) during the study
Secondary Outcome
Change from maralixibat baseline over the course of the study in the weekly average morning ItchRO(Obs)™ (Itch Reported Outcome) severity score
Condition
Progressive Familial Intrahepatic Cholestasis (PFIC)
Intervention
Maralixibat
Study Arms / Comparison Groups
Maralixibat
Description: All subjects will receive Maralixibat oral solution
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
30
Start Date
January 8, 2020
Completion Date
December 2022
Primary Completion Date
November 2022
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
Gender
All
Ages
1 Year - 18 Years
Accepts Healthy Volunteers
No
Contacts
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Location Countries
Argentina
Location Countries
Argentina
Administrative Informations
NCT ID
NCT04185363
Organization ID
MRX-503
Responsible Party
Sponsor
Study Sponsor
Mirum Pharmaceuticals, Inc.
Study Sponsor
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Verification Date
December 2021