Alagille Syndrome
Conditions
Brief summary
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.
Interventions
None listed
Sponsors
Ipsen
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Months to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosed with ALGS. * On (or starting) active odevixibat treatment. * Aged 6 months or older at the time of consent.
Exclusion criteria
* Currently participating in a clinical trial with odevixibat. * Currently participating in any interventional clinical trial for ALGS. * Have any contraindication to odevixibat as per the locally approved label.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of participants experiencing adverse events (AEs) | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment. |
| Percentage of participants experiencing serious adverse events (SAEs) | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | An adverse event (AE) is any untoward medical occurrence in a participant administered odevixibat, whether or not considered related to treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in fat-soluble vitamin (FSV) levels | From baseline and up to end of data collection (approximately 5 years of data collection) | — |
| Percentage of participants with fat-soluble vitamin deficiency | 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 | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | For example, bleeding, rickets, or osteopenia. |
| Percentage of participants with suspected hepatotoxicity requiring interruption of odevixibat treatment | 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 | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | — |
| Change from baseline in alanine aminotransferase (ALT) | From baseline and up to end of data collection (approximately 5 years of data collection) | — |
| Change from baseline in aspartate aminotransferase (AST) | From baseline and up to end of data collection (approximately 5 years of data collection) | — |
| Change from baseline in gamma-glutamyl transferase (GGT) | From baseline and up to end of data collection (approximately 5 years of data collection) | — |
| Change from baseline in blood bilirubin | From baseline and up to end of data collection (approximately 5 years of data collection) | — |
| Change from baseline in international normalized ratio (INR) | From baseline and up to end of data collection (approximately 5 years of data collection) | — |
| Percentage of participants with hospitalisations due to diarrhoea | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | — |
| Percentage of participants with liver transplantation | 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 | 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 | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | — |
| Percentage of participants with severe diarrhoea events | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | — |
| Percentage of participants with bloody diarrhoea events | 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 | 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 | From first ICF signature and up to end of data collection (approximately 5 years of data collection) | — |
Countries
France, Italy
Contacts
CONTACTIpsen Clinical Study Enquiries
STUDY_DIRECTORIpsen Medical Director
Ipsen
Outcome results
None listed