Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome in the European Union (LEAP-EU)

Summary

The key objectives of this low-intervention clinical study are to evaluate tolerability, long-term safety and long-term efficacy for patients with Alagille syndrome (ALGS) who are prescribed Livmarli.

Eligibility

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

Conditions3

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