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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. august 2026 oppdatert av: 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.

Studieoversikt

Status

Rekruttering

Forhold

Alagille syndrom

Studietype

Observasjonsmessig

Registrering (Antatt)

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Navn: Ipsen Clinical Study Enquiries
Telefonnummer: See e mail
E-post: clinical.trials@ipsen.com

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

Barn
Voksen
Eldre voksen

Tar imot friske frivillige

Nei

Prøvetakingsmetode

Ikke-sannsynlighetsprøve

Studiepopulasjon

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.

Beskrivelse

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.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

Hva måler studien?

Primære resultatmål

Resultatmål	Tiltaksbeskrivelse	Tidsramme
Percentage of participants experiencing adverse events (AEs) Tidsramme: 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) Tidsramme: 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)

Sekundære resultatmål

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Ipsen

Etterforskere

Studieleder: Ipsen Medical Director, Ipsen

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

10. juli 2026

Primær fullføring (Antatt)

30. september 2031

Studiet fullført (Antatt)

30. september 2031

Datoer for studieregistrering

Først innsendt

6. mai 2026

Først innsendt som oppfylte QC-kriteriene

6. mai 2026

Først lagt ut (Faktiske)

13. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

31. august 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

28. august 2026

Sist bekreftet

1. august 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

CLIN-60240-034

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

IPD-planbeskrivelse

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-delingstidsramme

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.

Tilgangskriterier for IPD-deling

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

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Resultatmål	Tiltaksbeskrivelse	Tidsramme
Percentage of participants with severe diarrhoea events Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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) Tidsramme: 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) Tidsramme: 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) Tidsramme: 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 Tidsramme: 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) Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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 Tidsramme: 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)