NCT07290257 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)
| NCT ID | NCT07290257 |
| Status | Recruiting |
| Phase | Phase 4 |
| Sponsor | Mirum Pharmaceuticals, Inc. |
| Condition | Alagille Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 230 participants |
| Start Date | 2025-09-25 |
| Primary Completion | 2030-09-22 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 4 studies follow an already-approved treatment in real-world conditions to monitor long-term safety and effectiveness.
This trial targets 230 participants in total. It began in 2025-09-25 with a primary completion date of 2030-09-22.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07290257 clinical trial?
This trial is open to participants of all sexes, aged 2 Months or older, studying Alagille Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07290257 trial and what does that mean for participants?
Phase 4 studies are conducted after a treatment has been approved. They monitor long-term safety and real-world effectiveness in a broader patient population.
Is NCT07290257 currently recruiting?
Yes, NCT07290257 is actively recruiting participants. Contact the research team at clinicaltrials@mirumpharma.com for enrollment information.
Where is the NCT07290257 trial being conducted?
This trial is being conducted at Brussels, Belgium, Ghent, Belgium, Bron, France, Toulouse, France and 11 additional locations.
Who is sponsoring the NCT07290257 clinical trial?
NCT07290257 is sponsored by Mirum Pharmaceuticals, Inc.. The trial plans to enroll 230 participants.