Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).

Phase 1

• Conditions: In patients with progressive familial intrahepatic cholestasis (PFIC),impairment of the egress of bile acids from the liver leads to cholestasis,hepatocellular injury and damage, and progressive liver disease thatmay ultimately lead to the need for liver transplantation. Itch is acommon symptom associated with cholestasis, it can occur at all stagesof cholestatic liver disease, with or without jaundice.; MedDRA version: 20.0Level: PTClassification code 10076033Term: Progressive familial intrahepatic cholestasisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2019-003395-39-AT
• Lead Sponsor: Mirum Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-01-08
• Last Update Posted: 13 November 2023

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

- Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee.
- 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.
- Males and females of nonchildbearing potential. Males and
nonpregnant, nonlactating females of childbearing potential who are
sexually active must agree to use acceptable methods of contraception
during the study through 30 days after the last dose of maralixibat.
- Females of childbearing potential must have a negative urine
pregnancy test result at the Baseline Visit (Day 0).
- Access to email or telephone for scheduled remote visits.
- Ability to read and understand the questionnaires (both caregivers and subjects above the age of assent).
- Access to consistent caregiver(s) during the study.
- Subject and caregiver willingness to comply with all study visits and requirements.

Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 noncompliance in study MRX-502, nonadherence to medical
regimens, unreliability, mental instability, or incompetence that could
compromise the validity of informed consent or lead to nonadherence
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 (including decompensated liver disease) 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.
- 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified