This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching. The main aim of this study is to observe the long-term, everyday safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.
Study Type
OBSERVATIONAL
Enrollment
30
Percentage of participants experiencing adverse events (AEs)
An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants experiencing serious adverse events (SAEs)
An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment.
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with severe diarrhoea events
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with bloody diarrhoea events
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants experiencing diarrhoea events with concurrent dehydration
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants experiencing diarrhoea events treated with oral or intravenous rehydration
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in fat-soluble vitamin (FSV) levels
Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)
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Percentage of participants with fat-soluble vitamin deficiency
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with clinical manifestations of fat-soluble vitamin deficiency
For example, bleeding, rickets, or osteopenia.
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with suspected hepatotoxicity requiring interruption of odevixibat treatment
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with clinical manifestations related to hepatotoxicity
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in alanine aminotransferase (ALT)
Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in aspartate aminotransferase (AST)
Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in gamma-glutamyl transferase (GGT)
Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in blood bilirubin
Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in international normalized ratio (INR)
Time frame: From baseline and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to diarrhoea
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to hepatotoxicity
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to fat-soluble vitamin deficiency
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with treatment discontinuations due to diarrhoea
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with treatment discontinuations due to hepatotoxicity
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with treatment discontinuations due to fat-soluble vitamin deficiency
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection).
Percentage of participants with pregnancy and maternal complications
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of foetuses, neonates, or infants with adverse effects following exposure to odevixibat during pregnancy and/or lactation
Time frame: From first documented exposure during pregnancy or lactation and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with biliary diversion surgery
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with liver transplantation
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants who die from any cause
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants switching from odevixibat to maralixibat
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)