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

Launched by MIRUM PHARMACEUTICALS, INC. · Dec 2, 2019

Keywords

ClinConnect Summary

This clinical trial is studying a medication called maralixibat for patients with a rare liver condition known as Progressive Familial Intrahepatic Cholestasis (PFIC). The main goal of this study is to look at how safe maralixibat is and how well patients can tolerate it over a longer period of time. This is an extension of a previous study, so participants must have completed that earlier study to be eligible for this one.

To join the trial, participants need to be between the ages of 1 and 18 years and provide consent or assent as required. It’s important to note that some individuals, such as pregnant or breastfeeding women, or those who have experienced serious side effects from maralixibat in the past, cannot participate. Throughout the trial, participants can expect regular check-ups and monitoring to ensure their safety. This study is currently active, but it is not recruiting new participants at this time.

Gender

ALL

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