A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
6 maggio 2026 aggiornato da: 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.
Panoramica dello studio
Stato
Non ancora reclutamento
Condizioni
Tipo di studio
Osservativo
Iscrizione (Stimato)
30
Contatti e Sedi
Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.
Contatto studio
- Nome: Ipsen Clinical Study Enquiries
- Numero di telefono: See e mail
- Email: clinical.trials@ipsen.com
Criteri di partecipazione
I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.
Criteri di ammissibilità
Età idonea allo studio
- Bambino
- Adulto
- Adulto più anziano
Accetta volontari sani
No
Metodo di campionamento
Campione non probabilistico
Popolazione di studio
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.
Descrizione
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.
Piano di studio
Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.
Come è strutturato lo studio?
Dettagli di progettazione
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Percentage of participants experiencing adverse events (AEs)
Lasso di tempo: 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)
Lasso di tempo: 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)
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Percentage of participants with severe diarrhoea events
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
|
Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Sponsor
Investigatori
- Direttore dello studio: Ipsen Medical Director, Ipsen
Studiare le date dei record
Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.
Studia le date principali
Inizio studio (Stimato)
30 settembre 2026
Completamento primario (Stimato)
30 settembre 2031
Completamento dello studio (Stimato)
30 settembre 2031
Date di iscrizione allo studio
Primo inviato
6 maggio 2026
Primo inviato che soddisfa i criteri di controllo qualità
6 maggio 2026
Primo Inserito (Effettivo)
13 maggio 2026
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
13 maggio 2026
Ultimo aggiornamento inviato che soddisfa i criteri QC
6 maggio 2026
Ultimo verificato
1 maggio 2026
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
- Malattia cardiovascolare
- Malattie genetiche, congenite
- Malattie dell'apparato digerente
- Malattie delle vie biliari
- Malattie del fegato
- Anomalie congenite
- Anomalie cardiovascolari
- Difetti cardiaci, congeniti
- Anomalie multiple
- Malattie del dotto biliare
- Colestasi, intraepatico
- Colestasi
- Malattie e anomalie congenite, ereditarie e neonatali
- Sindrome di Alagille
Altri numeri di identificazione dello studio
- CLIN-60240-034
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
SÌ
Descrizione del piano IPD
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.
Periodo di condivisione IPD
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.
Criteri di accesso alla condivisione IPD
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
No
Studia un dispositivo regolamentato dalla FDA degli Stati Uniti
No
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .