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)

Phase 3

Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC)
(PFIC)
T51.2

Registration Number: LBCTR2021034759

Lead Sponsor: Mirum Pharmaceuticals Inc

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Other

Sex: All

Target Recruitment: 90

Inclusion Criteria

1. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee.
2. Completion of study MRX-502; treatment interruption between MRX-502 and MRX-503 should be avoided. Subjects who do not complete the study MRX-503 Baseline Visit (Day 0) on the same day as the study MRX-502 EOT Visit will be considered for participation in study MRX-503 only after discussion with the Medical Monitor.
3. Males and females of non-childbearing potential. Males and non-pregnant, non-lactating females of childbearing potential who are sexually active must agree to use acceptable contraception during the study through 30 days after the last dose of maralixibat.
4. Females of childbearing potential must have a negative urine pregnancy test at the Baseline Visit (Day 0).
5. Access to email or telephone for scheduled remote visits.
6. Ability to read and understand the questionnaires (both caregivers and subjects above the age of assent).
7. Access to consistent caregiver(s) during the study.
8. Subject and caregiver willingness to comply with all study visits and requirements.

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.
6. Cognitive impairment of the subject or caregiver that would, in the opinion of the Investigator, preclude appropriate understanding of study information and compliance with study procedures.