Table of contents
- Trial overview
- Who is being studied
- What the trials measure
- Study design and phase
- Long-term follow-up and clinical outcomes
Trial overview
The available study is NCT07290257, a long-term study of MARALIXIBAT in people with Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille syndrome.[1] It is an interventional study, which means participants receive a treatment and the researchers collect results over time.[1]
The study is authorised and has an enrollment of 223 participants.[1] The trial title is “Long Term Study of Livmarli,” and the intervention listed is Livmarli 9.5 mg/mL oral solution given by mouth.[1]
Who is being studied
This trial focuses on people with PFIC and Alagille syndrome who are prescribed MARALIXIBAT.[1] These are liver diseases that can cause cholestasis, which means bile does not flow normally from the liver.[1]
The brief summary shows that the study is designed for participants with ALGS and for participants with PFIC, and the goals are slightly different for each group.[1] For ALGS, the study mainly evaluates tolerability, long-term safety, and long-term efficacy.[1] For PFIC, the study also looks at long-term clinical outcome events, growth and development, biomarkers, and pruritus over time.[1]
What the trials measure
The primary outcomes include safety and adverse events (AEs), which are unwanted medical problems that happen during the study.[1] The study also measures changes in liver function tests, serum bile acids, and markers related to vitamin K status, including FSV levels and INR.[1]
Another major outcome is pruritus severity, which means how bad the itching is.[1] Itching is measured using the Clinician Scratch Scale (CSS), and the study also tracks how often participants need other medicines for liver disease, itching, or cholestasis.[1]
The study also measures growth, including height or length and weight, both as raw values and as z-scores.[1] In simple terms, z-scores compare a child’s growth with what is expected for age and sex.[1]
Additional safety checks include dose reduction, treatment interruption, and treatment discontinuation because of adverse events or poor tolerability.[1] The study also records overdose, dosing errors, medication errors, misuse, and abuse frequencies.[1]
Study design and phase
This is a Phase 3 trial.[1] Phase 3 studies usually involve a larger number of participants and help researchers learn more about safety and how well a treatment works in the target population.[1]
The study is described as a low-intervention clinical study, meaning it is designed to collect long-term information in people already prescribed the treatment rather than to test a new experimental use from scratch.[1] The trial also follows starting dose and dose escalation, which means the treatment may be started at one dose and then increased over time while safety is watched closely.[1]
Long-term follow-up and clinical outcomes
For participants with PFIC, the trial looks at long-term clinical outcomes such as surgical biliary diversion, liver transplant, portal hypertension, complications of liver cirrhosis, liver carcinoma, disease progression, liver decompensation, liver-related mortality, and all-cause mortality.[1] These are important events that show how the liver disease changes over time.[1]
The study also tracks liver transplant waitlist status, which helps researchers understand how severe the disease becomes in some participants.[1] In addition, it collects biomarkers of cholestasis, liver fibrosis, and liver function, including APRI and PELD.[1]
For participants with PFIC, the summary also notes that possible liver toxicity will be monitored, and long-term safety of chronic exposure to propylene glycol will be evaluated.[1] This means the researchers are watching for any liver-related safety concerns and other long-term treatment effects.[1]
