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

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What is this study about?

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.

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 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 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 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 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 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 Study?

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 Study?

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.

Where you can join this trial?

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Other Sites

Site Name	City	Country
Hopital Des Enfants	Toulouse	France
Universitair Ziekenhuis Gent	Gent	Belgium
Universitaetsklinikum Regensburg AöR	Regensburg	Germany
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Istituto Mediterraneo Per I Trapianti E Terapie Ad Alta Specializzazione S.r.l.	Palermo	Italy
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hopital Beaujon	Clichy	France
Cmrfmvxbw Uhqhaokypxbqxn Sejrhpflh	Woluwe-Saint-Lambert	Belgium
Uxmntfowuu Myprano Chllzx Hqanesdbsowridtgf	Hamburg	Germany
Ucymkztenjqozndwpcrqj Ebfbh Als	Essen	Germany
Upwmtgpjitjl Mbrbskr Cjqypmb Gjomasrks	Groningen	The Netherlands
Aamplbx Ouucvrunbce Pgtu Giybljpx Xyodr	Bergamo	Italy
Fuzkkexwl Pkrh Lh Ikxzozmjuijqz Bnprppogh Dxd Hzwdotdz Unsegwlhwagnp Lt Pjh	Madrid	Spain
Hvdpxiil Vzga dwyhakhm	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	15.08.2025
France	Recruiting	15.08.2025
Germany	Recruiting	15.08.2025
Italy	Recruiting	15.08.2025
Spain	Recruiting	15.08.2025
The Netherlands	Recruiting	15.08.2025

Trial locations

Investigated drugs:

MARALIXIBAT

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.

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 ID:

2024-516804-40-00

Protocol code:

MRX-803

NCT ID:

NCT07290257

Trial Phase:

Therapeutic confirmatory (Phase III)

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

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