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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 de agosto de 2026 atualizado por: 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.

Visão geral do estudo

Status

Recrutamento

Condições

Tipo de estudo

Observacional

Inscrição (Estimado)

30

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Locais de estudo

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

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

  • Filho
  • Adulto
  • Adulto mais velho

Aceita Voluntários Saudáveis

Não

Método de amostragem

Amostra Não Probabilística

População do estudo

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.

Descrição

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.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Percentage of participants experiencing adverse events (AEs)
Prazo: 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)
Prazo: 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)

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Percentage of participants with severe diarrhoea events
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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)
Prazo: 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)
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Ipsen

Investigadores

  • Diretor de estudo: Ipsen Medical Director, Ipsen

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

10 de julho de 2026

Conclusão Primária (Estimado)

30 de setembro de 2031

Conclusão do estudo (Estimado)

30 de setembro de 2031

Datas de inscrição no estudo

Enviado pela primeira vez

6 de maio de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

6 de maio de 2026

Primeira postagem (Real)

13 de maio de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

31 de agosto de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

28 de agosto de 2026

Última verificação

1 de agosto de 2026

Mais Informações

Termos relacionados a este estudo

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • CLIN-60240-034

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

SIM

Descrição do plano 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.

Prazo de Compartilhamento de 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.

Critérios de acesso de compartilhamento 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/).

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

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