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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
230
Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.
Cliniques Universitaires Saint Luc (UCLouvain)
Brussels, Belgium
RECRUITINGUniversity Hospital Gent (UZ Gent)
Ghent, Belgium
RECRUITINGHospices Civils de Lyon - Hopital Femme Mère Enfant
Bron, Auvergne-Rhône-Alpes, France
RECRUITINGCHU de Toulouse - Hôpital des Enfants
Toulouse, Occitanie, France
RECRUITINGBicetre University Hospital
Le Kremlin-Bicêtre, Île-de-France Region, France
RECRUITINGCharite Berlin
Berlin, Germany
RECRUITINGChildren's University Hospital Essen
Essen, Germany
RECRUITINGUniversity Hospital Hamburg-Eppendorf
Hamburg, Germany
RECRUITING2nd Paediatric Clinic P &A Kyriakou Children's Hospital
Athens, Greece
NOT_YET_RECRUITINGAO Ospedale PAPA GIOVANNI XXIII
Bergamo, Lombardy, Italy
RECRUITING...and 5 more locations
Adverse Events (AEs)
Number and proportion of participants with AEs.
Time frame: Up to 7 days after the last dose of Livmarli.
Liver function tests (LFTs) - measuring enzyme activity or concentration
Change in LFTs from Baseline: Total and direct bilirubin, aminotransferases and GGT.
Time frame: Once every 3-4 months, for 5 years.
Fat-Soluble Vitamins (FSV) Concentration
Change in FSV levels (vitamins A, D, and E) from Baseline.
Time frame: Once every 3-4 months, for 5 years.
International Normalized Ratio (INR)
Change in INR level from Baseline.
Time frame: Once every 3-4 months, for 5 years.
Long-Term Clinical Outcomes
Number of participants with surgical biliary diversion, liver transplantation, liver transplant waitlist status change, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, liver decompensation, and death.
Time frame: Once every 3-4 months, for 5 years.
Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC
Change in PG level from Baseline.
Time frame: Once every 3-4 months, for 5 years.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.