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
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