Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients W… (NCT07290257) | Clinical Trial Compass
RecruitingPhase 4
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)
Belgium, France, Germany230 participantsStarted 2025-09-25
Plain-language summary
In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment.
In patients with Progressive Familial Intrahepatic Cholestasis (PFIC), the key objectives are to evaluate tolerability, long-term safety (including possible liver toxicity and potential impact of chronic exposure to propylene glycol (PG)), long-term efficacy, and growth and development.
Who can participate
Age range
2 Months
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Understand and execute an Informed consent and assent (as applicable)
* For participants with ALGS ≥2 months of age at Day 1
* For participants with PFIC, ≥3 months of age at Day 1
* A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis, or a clinically and/or genetically confirmed PFIC diagnosis
* For the ALGS primary cohort: Initiation of Livmarli at the time of study entry
* For the ALGS supplemental cohort, : Actively using Livmarli prior to study entry
* For participants with PFIC: Prescribed Livmarli at the time of study entry or prior to study entry
Exclusion Criteria:
* History of Liver Transplant
* Any Livmarli contraindications (as per SmPC)
* Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study
* Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded-access programs is acceptable.)
* Received another IBAT inhibitor within 7 days before the first dose of Livmarli
* Baseline data before start of treatment of Livmarli are unavailable (\<2 values before treatment) for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameter
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Adverse Events (AEs)
Timeframe: Up to 7 days after the last dose of Livmarli.
2
Liver function tests (LFTs) - measuring enzyme activity or concentration
Timeframe: Once every 3-4 months, for 5 years.
3
Liver function tests (LFTs) - measuring enzyme activity or concentration
Timeframe: Once every 3-4 months, for 5 years.
4
Fat-Soluble Vitamins (FSV) Concentration
Timeframe: Once every 3-4 months, for 5 years.
5
International Normalized Ratio (INR)
Timeframe: Once every 3-4 months, for 5 years.
6
Long-Term Clinical Outcomes
Timeframe: Once every 3-4 months, for 5 years.
7
Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC