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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

6. maj 2026 opdateret af: 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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Undersøgelsestype

Observationel

Tilmelding (Anslået)

30

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Prøveudtagningsmetode

Ikke-sandsynlighedsprøve

Studiebefolkning

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

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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

Resultatmål
Foranstaltningsbeskrivelse
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)

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Ipsen

Efterforskere

  • Studieleder: Ipsen Medical Director, Ipsen

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

30. september 2026

Primær færdiggørelse (Anslået)

30. september 2031

Studieafslutning (Anslået)

30. september 2031

Datoer for studieregistrering

Først indsendt

6. maj 2026

Først indsendt, der opfyldte QC-kriterier

6. maj 2026

Først opslået (Faktiske)

13. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

13. maj 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

6. maj 2026

Sidst verificeret

1. maj 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • CLIN-60240-034

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

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.

IPD-delingsadgangskriterier

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

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

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Ingen

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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Betingelser

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Placeringer

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