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

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

, , 

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

, , 


Verification Date

December 2021