Table of contents

• Trial overview
• Who is being studied
• What the trials measure
• Phase and study design
• Long-term follow-up and safety monitoring
• Main trial details

Trial overview

The available trial data describe one interventional study of MARALIXIBAT in people with progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS).^[1] The study is authorised and is listed as a Phase 3 trial with 223 participants.^[1]

The trial title is “Long Term Study of Livmarli,” and the intervention is an oral solution of Livmarli 9.5 mg/mL, given by mouth.^[1] The study is interventional, which means the researchers are giving the treatment and then measuring outcomes over time.^[1]

Who is being studied

The trial includes participants with PFIC and ALGS who are prescribed Livmarli.^[1] These are liver conditions linked to cholestasis, a problem where bile does not flow normally from the liver.^[1]

In the study summary, the PFIC group is followed for long-term safety, possible liver toxicity, and long-term clinical outcomes such as surgical biliary diversion, liver transplant, portal hypertension, complications of cirrhosis, liver carcinoma, disease progression, liver decompensation, liver-related mortality, and all-cause mortality.^[1] The ALGS group is followed mainly for tolerability, long-term safety, and long-term efficacy.^[1]

What the trials measure

The primary outcomes include safety, efficacy, and tolerability.^[1] Safety is checked by counting adverse events, which are unwanted medical events during the study, and by looking at changes in liver function tests.^[1]

The study also measures changes in pruritus, which means itching, using the Clinician Scratch Scale (CSS).^[1] Other measures include changes in serum bile acids, use of other medicines for liver disease, itching, or cholestasis, and the timing of long-term clinical outcomes.^[1]

For participants with PFIC, the study also monitors PG toxicity, meaning possible problems linked to chronic exposure to propylene glycol.^[1] Growth is another endpoint, with height, length, and weight measured as absolute values and z-scores.^[1] The trial also tracks overdose, dosing errors, misuse, and abuse frequencies.^[1]

Phase and study design

This is a Phase 3 study, which is a later stage of clinical research in a larger group of participants.^[1] The study type is interventional, so the treatment is part of the research plan rather than only being observed in routine care.^[1]

The study is described as a low-intervention clinical study in the summary, meaning the researchers are not adding many extra treatment steps beyond the planned use of Livmarli.^[1] The trial is designed to learn how the treatment performs over a long period rather than only for short-term results.^[1]

Long-term follow-up and safety monitoring

A major focus of the trial is long-term follow-up.^[1] The study looks at how often participants need dose reduction, treatment interruption, or treatment discontinuation because of adverse events or poor tolerability.^[1]

For PFIC, the trial also tracks long-term clinical outcome events such as surgical biliary diversion, liver transplant, portal hypertension, cirrhosis complications, liver carcinoma, disease progression, liver decompensation, liver-related mortality, and all-cause mortality.^[1] The study also evaluates liver transplant waitlist status, which means whether a participant is waiting for a transplant.^[1]

Additional biomarker data are collected for cholestasis, liver fibrosis, and liver function, including APRI and PELD.^[1] Biomarkers are lab measurements that help show what is happening in the body.^[1]

Main trial details

The following table gives the key details from the trial record.^[1]