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Study on Long-Term Safety and Effects of Maralixibat for Patients with Alagille Syndrome

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Alagille syndrome, a rare genetic disorder that affects the liver and other parts of the body. The study will use a treatment called Livmarli, which is an oral solution containing the active substance maralixibat. The purpose of the study is to evaluate how well patients tolerate the treatment, its long-term safety, and its effectiveness in managing symptoms of Alagille syndrome.

Participants in the study will take Livmarli as prescribed, and researchers will monitor them over an extended period. The study will look at various aspects, such as any side effects experienced, changes in liver function, and the severity of itching, which is a common symptom of the disease. The study will also observe how the treatment affects the levels of certain substances in the blood and the use of other medications for liver disease.

The trial aims to gather information on the safety of Livmarli over a long period, including any potential liver-related issues. It will also assess how well patients can tolerate the starting dose and any changes in dosage. The study will continue until 2035, providing valuable insights into the long-term management of Alagille syndrome with Livmarli.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, you will be required to provide informed consent. This means you agree to participate after understanding the study's purpose and procedures.

    If applicable, you may also need to provide assent, which is a form of agreement for participants who are not legally able to give full consent.

  2. Step 2

    Eligibility confirmation

    Your eligibility will be confirmed based on a clinically and/or genetically confirmed diagnosis of Alagille syndrome, with itching (pruritus) due to chronic liver issues.

    You must be prescribed Livmarli at the time of joining the study or have been prescribed it before joining.

  3. Step 3

    Medication administration

    You will take Livmarli 9.5 mg/mL oral solution as part of the study. This medication is taken by mouth.

    The dosage and frequency will be determined by your healthcare provider, and you will be monitored for any side effects or changes in your condition.

  4. Step 4

    Monitoring and assessments

    Throughout the study, you will undergo regular assessments to monitor the safety and efficacy of the treatment. This includes checking for any adverse events and changes in liver function.

    Your healthcare provider will also assess the severity of your itching and any changes in bile acid levels in your blood.

  5. Step 5

    Tolerability evaluation

    The study will evaluate how well you tolerate the starting dose and any dose increases. This involves summarizing any adverse events or other safety information.

    If necessary, adjustments to your dose may be made, including reducing the dose, pausing treatment, or stopping it altogether if you experience poor tolerability.

  6. Step 6

    Long-term follow-up

    The study is designed to continue until April 19, 2035, allowing for long-term observation of your condition and the effects of the treatment.

    Regular follow-ups will help track your progress and any long-term outcomes related to your liver health and overall well-being.

Who can join the trial?

8 criteria

  • Provide informed consent and assent if applicable. This means you understand the study and agree to participate.
  • Have a confirmed diagnosis of Alagille syndrome (ALGS), either through clinical evaluation or genetic testing. ALGS is a genetic disorder that can affect the liver and other parts of the body.
  • Experience pruritus due to chronic cholestasis. Pruritus is a medical term for itching, and chronic cholestasis refers to a long-term condition where bile flow from the liver is reduced or blocked.
  • For the primary group, you must be prescribed Livmarli at the time you join the study. Livmarli is a medication used to treat symptoms of ALGS.
  • For the additional group, you must have been prescribed Livmarli before joining the study.
  • Be within the age range of 2 to 18 years old.
  • Both male and female participants are eligible.
  • The study includes individuals who may be considered part of a vulnerable population. This means people who might need special protection or care.

Who cannot join the trial?

5 criteria

  • Patients who do not have **Alagille syndrome** cannot participate. Alagille syndrome is a genetic disorder that affects the liver and other parts of the body.
  • Patients who are not within the age range of **2 to 4 years old** cannot participate.
  • Patients who are not part of the specified clinical trial group cannot participate.
  • Both **male and female** patients are eligible, so gender is not a reason for exclusion.
  • Patients who are not considered part of a **vulnerable population** cannot participate. A vulnerable population includes groups who may have additional protection needs in a clinical trial.
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Investigated drugs

Livmarli is a medication being studied for its effects on patients with Alagille Syndrome, a genetic disorder that can affect the liver and other parts of the body. The main goal of this study is to see how well patients tolerate Livmarli over a long period, to check for any potential liver-related side effects, and to understand how effective it is in treating the symptoms of Alagille Syndrome. This study is important because it helps to gather information on the safety and benefits of using Livmarli for a longer time in people with this condition.

What is already known about the treatment

Livmarli – Livmarli is administered orally, meaning it is taken by mouth, and is currently being studied for its long-term safety and effectiveness in treating Alagille syndrome, a rare genetic disorder affecting the liver. This medication is in a clinical trial phase in the European Union, focusing on its tolerability and potential liver toxicity. Livmarli is primarily used to manage symptoms associated with Alagille syndrome, such as liver-related issues. It works at the molecular level by targeting specific pathways in the liver to help reduce bile acid buildup, which can alleviate symptoms and improve liver function. Livmarli is classified pharmacologically as a bile acid modulator.

Investigated diseases

Alagille syndrome – Alagille syndrome is a genetic disorder that primarily affects the liver and heart, but it can also impact other parts of the body. It is characterized by a reduced number of bile ducts in the liver, leading to bile buildup and liver damage. Over time, this can cause symptoms such as jaundice, itching, and poor growth. The condition may also involve heart defects, distinctive facial features, and abnormalities in the eyes, spine, and kidneys. As the disease progresses, liver function may decline, and other organs may be increasingly affected. The severity and progression of symptoms can vary widely among individuals with Alagille syndrome.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-516804-40-00Protocol codeMRX-803Estimated enrolment231 patientsSponsorMirum Pharmaceuticals Inc.

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