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

28 augustus 2026 bijgewerkt door: Ipsen

Prospective Non-Interventional Study Evaluating the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS)

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.

Studie Overzicht

Toestand

Werving

Conditie

Alagille-syndroom

Studietype

Observationeel

Inschrijving (Geschat)

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Naam: Ipsen Clinical Study Enquiries
Telefoonnummer: See e mail
E-mail: clinical.trials@ipsen.com

Studie Locaties

Frankrijk
- - Bron, Frankrijk
    - Nog niet aan het werven
    - Hospices Civils de Lyon - Hopital Femme Mere Enfant
  - Dijon, Frankrijk
    - Nog niet aan het werven
    - CHU Dijon Bourgogne
  - Le Kremlin-Bicêtre, Frankrijk
    - Nog niet aan het werven
    - APHP - Hopital Bicetre - Paris Sud
  - Marseille, Frankrijk
    - Nog niet aan het werven
    - APHM - CHU Timone
  - Montpellier, Frankrijk
    - Nog niet aan het werven
    - CHU de Montpellier - Hopital Saint Eloi
  - Paris, Frankrijk
    - Nog niet aan het werven
    - APHP - Hopital Necker-Enfants Malades
  - Toulouse, Frankrijk
    - Nog niet aan het werven
    - CHU de Toulouse - Hopital Paule de Viguier
  - Villejuif, Frankrijk
    - Nog niet aan het werven
    - APHP - Centre Hepato-Biliaire Hospital Paul Brousse
Italië
- - Florence, Italië
    - Werving
    - Azienda Ospedaliero Universitaria Meyer
  - Naples, Italië
    - Nog niet aan het werven
    - Azienda Ospedaliera di Rilievo Nazionale Santobono Pausilipon
  - Padova, Italië
    - Nog niet aan het werven
    - University Hospital Of Padova
  - Roma, Italië
    - Nog niet aan het werven
    - Irccs Ospedale Pediatrico Bambino Gesu
  - Torino, Italië
    - Nog niet aan het werven
    - A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita
  - Udine, Italië
    - Nog niet aan het werven
    - Ospedale Universitario Di Udine

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

Kind
Volwassen
Oudere volwassene

Accepteert gezonde vrijwilligers

Nee

Bemonsteringsmethode

Niet-waarschijnlijkheidssteekproef

Studie Bevolking

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.

Beschrijving

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.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat	Maatregel Beschrijving	Tijdsspanne
Percentage of participants experiencing adverse events (AEs) Tijdsspanne: 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.	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) Tijdsspanne: 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.	From first ICF signature and up to end of data collection (approximately 5 years of data collection)

Secundaire uitkomstmaten

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Ipsen

Onderzoekers

Studie directeur: Ipsen Medical Director, Ipsen

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

10 juli 2026

Primaire voltooiing (Geschat)

30 september 2031

Studie voltooiing (Geschat)

30 september 2031

Studieregistratiedata

Eerst ingediend

6 mei 2026

Eerst ingediend dat voldeed aan de QC-criteria

6 mei 2026

Eerst geplaatst (Werkelijk)

13 mei 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

31 augustus 2026

Laatste update ingediend die voldeed aan QC-criteria

28 augustus 2026

Laatst geverifieerd

1 augustus 2026

Meer informatie

Termen gerelateerd aan deze studie

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

CLIN-60240-034

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

Beschrijving IPD-plan

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.

IPD-tijdsbestek voor delen

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.

IPD-toegangscriteria voor delen

Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Nee

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

Uitkomstmaat	Maatregel Beschrijving	Tijdsspanne
Percentage of participants with severe diarrhoea events Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with bloody diarrhoea events Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From baseline and up to end of data collection (approximately 5 years of data collection)		From baseline and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with fat-soluble vitamin deficiency Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)	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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in alanine aminotransferase (ALT) Tijdsspanne: From baseline and up to end of data collection (approximately 5 years of data collection)		From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in aspartate aminotransferase (AST) Tijdsspanne: From baseline and up to end of data collection (approximately 5 years of data collection)		From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in gamma-glutamyl transferase (GGT) Tijdsspanne: From baseline and up to end of data collection (approximately 5 years of data collection)		From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in blood bilirubin Tijdsspanne: From baseline and up to end of data collection (approximately 5 years of data collection)		From baseline and up to end of data collection (approximately 5 years of data collection)
Change from baseline in international normalized ratio (INR) Tijdsspanne: From baseline and up to end of data collection (approximately 5 years of data collection)		From baseline and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with hospitalisations due to diarrhoea Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection).		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection).		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection).		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first documented exposure during pregnancy or lactation and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Percentage of participants with liver transplantation Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		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 Tijdsspanne: From first ICF signature and up to end of data collection (approximately 5 years of data collection)		From first ICF signature and up to end of data collection (approximately 5 years of data collection)