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A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China

28. august 2026 oppdatert av: Ipsen

Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay).

This registry-based study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) who take odevixibat (Bylvay) as part of routine clinical care in China.

PFIC is a rare genetic liver disease that affects bile secretion and can cause bile acids to build up in the liver, which may lead to symptoms such as severe itching (pruritus).

Odevixibat was first allowed to be used for PFIC in babies older than 6 months by the European Medicines Agency (EMA) on 16 July 2021 and by the United States Food and Drug Administration (FDA) on 20 July 2021 for itching in babies older than 3 months. Odevixibat is approved for the treatment of pruritus in PFIC and was approved in China on 01 December 2024 for patients 6 months of age and older with PFIC.

The main aim of this registry is to assess long-term real-world safety (based on adverse events) and to describe effectiveness outcomes.

Studieoversikt

Status

Rekruttering

Forhold

Studietype

Observasjonsmessig

Registrering (Antatt)

20

Kontakter og plasseringer

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

Studiekontakt

Studiesteder

  • Kina
      • Beijing, Kina
        • Aktiv, ikke rekrutterende
        • Capital Children's Medical Center
      • Chongqing, Kina
        • Aktiv, ikke rekrutterende
        • Chongqing Medical University - Children's Hospital
      • Hunan, Kina
        • Rekruttering
        • Hunan Children's Hospital
      • Nanjing, Kina
        • Aktiv, ikke rekrutterende
        • Nanjing Medical University-Nanjing Children's Hospital
      • Shanghai, Kina
        • Rekruttering
        • Children's Hospital of Fudan University Endocrinology and Metabolism
      • Xiamen, Kina
        • Aktiv, ikke rekrutterende
        • Xiamen Children's Hospital

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 Progressive Familial Intrahepatic Cholestasis (PFIC) (all types) who have been prescribed odevixibat by their treating physician will be eligible. Participants who started odevixibat treatment before the implementation of the registry may also be enrolled.

Beskrivelse

Inclusion Criteria:

  • Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enroll the participant in this registry) by their treating physician
  • On (or starting) active odevixibat treatment (participants can remain in the registry during odevixibat treatment interruptions)
  • Signed informed consent and assent, as appropriate

Exclusion Criteria:

  • Currently participating in a clinical trial with odevixibat
  • Currently participating in any interventional clinical trial for PFIC
  • Have any contraindication to odevixibat as per the approved label in China

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), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment
From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
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), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
SAEs are collected as part of safety reporting and include events meeting seriousness criteria (e.g., death, life-threatening, hospitalization, etc.) as defined in the protocol
From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Event-free survival (EFS)
Tidsramme: From first ICF signature and up to end of data collection (approximately 5 years of data collection
EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death.
From first ICF signature and up to end of data collection (approximately 5 years of data collection
Surgical biliary diversion-free survival
Tidsramme: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Liver transplant-free survival
Tidsramme: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Overall survival
Tidsramme: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Defined as time from the start of odevixibat treatment to death.
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Pruritus improvement
Tidsramme: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Pruritus improvement described at each patient visit using a (semi-)objective scoring scale to assess level of pruritus from the start of the odevixibat treatment.
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in serum bile acid
Tidsramme: From baseline and up to end of data collection (approximately 5 years of data collection
Change from baseline assessed by measuring serum bile acid levels at each patient visit.
From baseline and up to end of data collection (approximately 5 years of data collection

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)

16. april 2026

Primær fullføring (Antatt)

30. april 2031

Studiet fullført (Antatt)

30. april 2031

Datoer for studieregistrering

Først innsendt

8. mai 2026

Først innsendt som oppfylte QC-kriteriene

14. mai 2026

Først lagt ut (Faktiske)

15. 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-60240-032

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (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

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

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