What the trial was testing
The MARCH-PFIC enrolled 93 patients with liver disease. The study was sponsored by Mirum Pharmaceuticals, Inc. and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Children on maralixibat saw their severe itching improve by an additional 1.1 points compared to placebo.
The lancet. Gastroenterology & hepatology · 2024 · NCT03905330
These findings — that children taking maralixibat had significantly less severe itching than those on placebo — were published in the The lancet. Gastroenterology & hepatology and represent the headline result of the study.
Researchers tracked outcomes across 93 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with liver disease, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Maralixibat received FDA approval in 2021 for PFIC. If your child has PFIC with severe itching, talk to your hepatologist about whether maralixibat could help. This was a large-scale study showing the medicine reduces itching and bile acids that damage the liver, potentially delaying the need for surgery or transplant.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open liver disease trials
JS207 Combined With JS007 as First-line Treatment for Advanced Hepatocellular Carcinoma
This open-label, multicenter phase II study was designed to evaluate the safety and efficacy of JS207 alone or in combination with JS007 as first-line treatment in subjects with advanced HCC. The subjects were all subjects with unresectable, locally advanced, recurrent, or metastatic HCC who had not received previous systemic therapy. Planned to enroll 43 to 72 subjects.The study was divided into two stages, dose exploration stage (3-12 cases) and random expansion stage (40-60 cases).After the dose exploration phase, a dose group was selected to enter the randomized expansion phase based on comprehensive discussion and evaluation of DLT, AE, dose adjustment, PK parameters and preliminary efficacy results in each dose group.
"Referral Attivo" Delta, Dallo Screening Alla Presa in Carico.
The aim of the "Active Delta Referral" Project is to estimate the prevalence of anti-HDV positivity among HBsAg-positive patients hospitalized in the Department of Medicine at ASST Papa Giovanni XXIII in Bergamo, as well as among patients at the SerD and the Prison Healthcare Unit of the same ASST, following the introduction of the new methodology in 2025, and to compare these findings with existing estimates for outpatients.