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)

Обзор

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.

Подробное описание

This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with either ALGS with cholestatic pruritus or PFIC who are treated with Livmarli.

Participants will be treated according to standard of care for the duration of the study.

Вмешательства

• Препарат Livmarli Oral Product
  Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.

Первичные конечные точки

• Adverse Events (AEs) [Срок оценки: Up to 7 days after the last dose of Livmarli.]
• Liver function tests (LFTs) - measuring enzyme activity or concentration [Срок оценки: Once every 3-4 months, for 5 years.]
• Liver function tests (LFTs) - measuring enzyme activity or concentration [Срок оценки: Once every 3-4 months, for 5 years.]
• Fat-Soluble Vitamins (FSV) Concentration [Срок оценки: Once every 3-4 months, for 5 years.]
• International Normalized Ratio (INR) [Срок оценки: Once every 3-4 months, for 5 years.]
• Long-Term Clinical Outcomes [Срок оценки: Once every 3-4 months, for 5 years.]
• Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC [Срок оценки: Once every 3-4 months, for 5 years.]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Другое

Центры проведения

Франция · 3 центра

• Hospices Civils de Lyon - Hopital Femme Mère Enfant — Bron
• CHU de Toulouse - Hôpital des Enfants — Toulouse
• Bicetre University Hospital — Le Kremlin-Bicêtre

Германия · 3 центра

• Charite Berlin — Berlin
• Children's University Hospital Essen — Essen
• University Hospital Hamburg-Eppendorf — Hamburg

Бельгия · 2 центра

• Cliniques Universitaires Saint Luc (UCLouvain) — Brussels
• University Hospital Gent (UZ Gent) — Ghent

Италия · 2 центра

• AO Ospedale PAPA GIOVANNI XXIII — Bergamo
• Istituto mediterraneo trapianti - ISMETT — Palermo

Испания · 2 центра

• Hospital Universitairo Vall D'Hebron — Barcelona
• Hospital Universitario La Paz — Madrid

Греция · 1 центр

• 2nd Paediatric Clinic P &A Kyriakou Children's Hospital — Athens

Нидерланды · 1 центр

• University Medical Center Groningen (UMCG) — Groningen

Португалия · 1 центр

• Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo — Coimbra

Идентификаторы

NCT: NCT07290257 · MRX-803

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗