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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.
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | From first ICF signature and up to end of data collection (approximately 5 years of data collection) |
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
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Inclusion Criteria:
Exclusion Criteria:
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Participants with Alagille syndrome (ALGS) who have been prescribed odevixibat by their treating physician will be enrolled into the study. Participants who started odevixibat treatment before study implementation may also be enrolled.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Ipsen Clinical Study Enquiries | Contact | See e mail | clinical.trials@ipsen.com |
| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical Director | Ipsen | Study Director |
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Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.
Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
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Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
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| ID | Term |
|---|---|
| D016738 | Alagille Syndrome |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
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| 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 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) |
| 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 | For example, bleeding, rickets, or osteopenia. | 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 | 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 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 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) |
| D004066 |
| Digestive System Diseases |
| D008107 | Liver Diseases |
| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |