Long-term safety and outcomes of Livmarli for Alagille syndrome in the EU
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)
This study will follow people with Alagille syndrome who take Livmarli to see if it is safe, well tolerated over the long term, and continues to reduce cholestatic itching.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | 2 Months and up |
| Sex | All |
| Sponsor | Mirum Pharmaceuticals, Inc. Industry-sponsored |
| Locations | 13 sites (Brussels and 12 other locations) |
| Trial ID | NCT07290257 on ClinicalTrials.gov |
What this trial studies
This is a multicenter, open-label, low-intervention study enrolling participants with clinically or genetically confirmed Alagille syndrome who have cholestatic pruritus and are prescribed Livmarli. Participants remain on standard-of-care treatment and are followed with scheduled safety and efficacy assessments, including liver function tests, fat-soluble vitamin labs, serum bile acids, and pruritus measures. The study includes a primary cohort of participants prescribed Livmarli at study entry and a supplemental cohort of those prescribed the drug prior to entry. Data collection focuses on long-term tolerability, safety outcomes, and sustained clinical effect on itching.
Who should consider this trial
Good fit: Ideal candidates are people aged 2 months or older with clinically or genetically confirmed Alagille syndrome who have cholestatic pruritus and are prescribed Livmarli either at or before study entry, and who can provide consent/assent.
Not a fit: Patients with a prior liver transplant, contraindications to Livmarli, insufficient baseline safety/efficacy data, or other conditions that would interfere with participation are unlikely to benefit from joining this study.
Why it matters
Potential benefit: If successful, the study could confirm that Livmarli is safe and well tolerated long term and continue to provide relief from cholestatic itching in people with Alagille syndrome.
How similar studies have performed: Previous clinical programs of maralixibat (Livmarli) in Alagille syndrome have shown improvements in pruritus and biochemical markers, supporting the rationale for this long-term safety follow-up.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Informed consent and assent (as applicable) * ≥2 months of age at Day 1 * A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis * For the primary cohort, prescribed Livmarli at time of study entry * For the supplemental cohort, prescribed Livmarli prior to study entry Exclusion Criteria: * History of Liver Transplant * Any contraindications against Livmarli (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.) * 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
Where this trial is running
Brussels and 12 other locations
- Cliniques Universitaires Saint Luc (UCLouvain) — Brussels, Belgium (Not_yet_recruiting)
- University Hospital Gent (UZ Gent) — Ghent, Belgium (Not_yet_recruiting)
- Hospices Civils de Lyon - Hopital Femme Mère Enfant — Bron, Auvergne-Rhône-Alpes, France (Recruiting)
- CHU de Toulouse - Hôpital des Enfants — Toulouse, Occitanie, France (Recruiting)
- Bicetre University Hospital — Le Kremlin-Bicêtre, Île-de-France Region, France (Recruiting)
- Charite Berlin — Berlin, Germany (Not_yet_recruiting)
- Children's University Hospital Essen — Essen, Germany (Not_yet_recruiting)
- University Hospital Hamburg-Eppendorf — Hamburg, Germany (Not_yet_recruiting)
- AO Ospedale PAPA GIOVANNI XXIII — Bergamo, Lombardy, Italy (Recruiting)
- Istituto mediterraneo trapianti - ISMETT — Palermo, Sicily, Italy (Not_yet_recruiting)
- University Medical Center Groningen (UMCG) — Groningen, Netherlands (Not_yet_recruiting)
- Hospital Universitairo Vall D'Hebron — Barcelona, Catalonia, Spain (Recruiting)
- Hospital Universitario La Paz — Madrid, Spain (Recruiting)
Study contacts
- Study coordinator: Clinical Trials Mirum
- Email: clinicaltrials@mirumpharma.com
- Phone: +16506674085
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.