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)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Livmarli Oral Product.
- Who it may be relevant to
- Registry conditions: Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis. Basic parameters: from 2 months · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Belgium, France, Germany, Greece, Italy +3
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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.
Detailed description
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.
Interventions
- Drug Livmarli Oral Product
Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.
Primary outcome measures
- Adverse Events (AEs) [Time frame: Up to 7 days after the last dose of Livmarli.]
- Liver function tests (LFTs) - measuring enzyme activity or concentration [Time frame: Once every 3-4 months, for 5 years.]
- Liver function tests (LFTs) - measuring enzyme activity or concentration [Time frame: Once every 3-4 months, for 5 years.]
- Fat-Soluble Vitamins (FSV) Concentration [Time frame: Once every 3-4 months, for 5 years.]
- International Normalized Ratio (INR) [Time frame: Once every 3-4 months, for 5 years.]
- Long-Term Clinical Outcomes [Time frame: Once every 3-4 months, for 5 years.]
- Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC [Time frame: Once every 3-4 months, for 5 years.]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Other
Study locations
France · 3 centers
- Hospices Civils de Lyon - Hopital Femme Mère Enfant — Bron
- CHU de Toulouse - Hôpital des Enfants — Toulouse
- Bicetre University Hospital — Le Kremlin-Bicêtre
Germany · 3 centers
- Charite Berlin — Berlin
- Children's University Hospital Essen — Essen
- University Hospital Hamburg-Eppendorf — Hamburg
Belgium · 2 centers
- Cliniques Universitaires Saint Luc (UCLouvain) — Brussels
- University Hospital Gent (UZ Gent) — Ghent
Italy · 2 centers
- AO Ospedale PAPA GIOVANNI XXIII — Bergamo
- Istituto mediterraneo trapianti - ISMETT — Palermo
Spain · 2 centers
- Hospital Universitairo Vall D'Hebron — Barcelona
- Hospital Universitario La Paz — Madrid
Greece · 1 center
- 2nd Paediatric Clinic P &A Kyriakou Children's Hospital — Athens
Netherlands · 1 center
- University Medical Center Groningen (UMCG) — Groningen
Portugal · 1 center
- Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo — Coimbra
Identifiers
NCT: NCT07290257 · MRX-803