CompletedPhase 3

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

United States90 participantsStarted 2020-01-08

Plain-language summary

The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.

Age range1 Year – 18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
✓. Completion of study MRX-502

✕. 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 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
✕. 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 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

Incidence of Treatment Emergent Adverse Events (TEAEs) during the study

Timeframe: From baseline through time of interim analysis, up to 120 weeks

NCT IDNCT04185363

SponsorMirum Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-04-23