In short
Clinical trials are investigating MARALIXIBAT in children and young people with Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille syndrome. These studies mainly look at long-term safety, tolerability, and how well treatment helps with itching, liver tests, growth, and other health outcomes.
Key points
- Clinical trials of MARALIXIBAT are studying children and young people with Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille syndrome. The main study is a Phase 3, long-term trial with 223 participants. It focuses on safety, tolerability, itching, liver tests, bile acids, growth, and long-term clinical outcomes. The trial also tracks the use of other medicines and events such as liver transplant, portal hypertension, and disease progression. Overall, the research aims to learn how MARALIXIBAT performs over time in people with these chronic liver conditions.
Trial overview
The available study is NCT07290257, a long-term study of MARALIXIBAT in people with Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille syndrome. It is an interventional study, which means participants receive a treatment and the researchers collect results over time.
The study is authorised and has an enrollment of 223 participants. The trial title is “Long Term Study of Livmarli,” and the intervention listed is Livmarli 9.5 mg/mL oral solution given by mouth.
Who is being studied
This trial focuses on people with PFIC and Alagille syndrome who are prescribed MARALIXIBAT. These are liver diseases that can cause cholestasis, which means bile does not flow normally from the liver.
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. For ALGS, the study mainly evaluates tolerability, long-term safety, and long-term efficacy. For PFIC, the study also looks at long-term clinical outcome events, growth and development, biomarkers, and pruritus over time.
What the trials measure
The primary outcomes include safety and adverse events (AEs), which are unwanted medical problems that happen during the study. The study also measures changes in liver function tests, serum bile acids, and markers related to vitamin K status, including FSV levels and INR.
Another major outcome is pruritus severity, which means how bad the itching is. 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.
The study also measures growth, including height or length and weight, both as raw values and as z-scores. In simple terms, z-scores compare a child’s growth with what is expected for age and sex.
Additional safety checks include dose reduction, treatment interruption, and treatment discontinuation because of adverse events or poor tolerability. The study also records overdose, dosing errors, medication errors, misuse, and abuse frequencies.
Study design and phase
This is a Phase 3 trial. 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.
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. 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.
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. These are important events that show how the liver disease changes over time.
The study also tracks liver transplant waitlist status, which helps researchers understand how severe the disease becomes in some participants. In addition, it collects biomarkers of cholestasis, liver fibrosis, and liver function, including APRI and PELD.
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. This means the researchers are watching for any liver-related safety concerns and other long-term treatment effects.