A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

Ipsen30 enrolled

Overview

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

Conditions

Alagille Syndrome

Eligibility

Sex: ALLMin age: 6 Months

Medical Language ↔ Plain English

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.

Locations (14)

Hospices Civils de Lyon - Hopital Femme Mere Enfant

Bron, France

NOT_YET_RECRUITING

CHU Dijon Bourgogne

Dijon, France

Outcomes

Primary Outcomes

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)

Secondary Outcomes

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)

Central Contacts

Ipsen Clinical Study Enquiries

CONTACT

See e mail clinical.trials@ipsen.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

APHP - Hopital Bicetre - Paris Sud

Le Kremlin-Bicêtre, France

NOT_YET_RECRUITING

APHM - CHU Timone

Marseille, France

NOT_YET_RECRUITING

CHU de Montpellier - Hopital Saint Eloi

Montpellier, France

NOT_YET_RECRUITING

APHP - Hopital Necker-Enfants Malades

Paris, France

NOT_YET_RECRUITING

CHU de Toulouse - Hopital Paule de Viguier

Toulouse, France

NOT_YET_RECRUITING

APHP - Centre Hepato-Biliaire Hospital Paul Brousse

Villejuif, France

NOT_YET_RECRUITING

Azienda Ospedaliero Universitaria Meyer

Florence, Italy

RECRUITING

Azienda Ospedaliera Di Rilievo Nazionale Santobono Pausilipon

Naples, Italy

NOT_YET_RECRUITING

...and 4 more locations

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)