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
14. Mai 2026 aktualisiert von: 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.
Studienübersicht
Status
Rekrutierung
Bedingungen
Studientyp
Beobachtungs
Einschreibung (Geschätzt)
20
Kontakte und Standorte
Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.
Studienkontakt
- Name: Ipsen Clinical Study Enquiries
- Telefonnummer: See e-mail
- E-Mail: clinical.trials@ipsen.com
Studienorte
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Shanghai, China
- Rekrutierung
- Children's Hospital of Fudan University Endocrinology and Metabolism
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Teilnahmekriterien
Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.
Zulassungskriterien
Studienberechtigtes Alter
- Kind
- Erwachsene
- Älterer Erwachsener
Akzeptiert gesunde Freiwillige
Nein
Probenahmeverfahren
Nicht-Wahrscheinlichkeitsprobe
Studienpopulation
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.
Beschreibung
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
Studienplan
Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.
Wie ist die Studie aufgebaut?
Designdetails
Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
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Percentage of participants experiencing adverse events (AEs)
Zeitfenster: 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.
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An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment
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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.
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Percentage of participants experiencing serious adverse events (SAEs)
Zeitfenster: 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.
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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
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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.
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Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
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Event-free survival (EFS)
Zeitfenster: From first ICF signature and up to end of data collection (approximately 5 years of data collection
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EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection
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Surgical biliary diversion-free survival
Zeitfenster: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Liver transplant-free survival
Zeitfenster: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Overall survival
Zeitfenster: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Defined as time from the start of odevixibat treatment to death.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Pruritus improvement
Zeitfenster: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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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.
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From first ICF signature and up to end of data collection (approximately 5 years of data collection)
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Change from baseline in serum bile acid
Zeitfenster: From baseline and up to end of data collection (approximately 5 years of data collection
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Change from baseline assessed by measuring serum bile acid levels at each patient visit.
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From baseline and up to end of data collection (approximately 5 years of data collection
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Mitarbeiter und Ermittler
Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.
Sponsor
Ermittler
- Studienleiter: Ipsen Medical Director, Ipsen
Studienaufzeichnungsdaten
Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.
Haupttermine studieren
Studienbeginn (Tatsächlich)
16. April 2026
Primärer Abschluss (Geschätzt)
30. April 2031
Studienabschluss (Geschätzt)
30. April 2031
Studienanmeldedaten
Zuerst eingereicht
8. Mai 2026
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
14. Mai 2026
Zuerst gepostet (Tatsächlich)
15. Mai 2026
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
15. Mai 2026
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
14. Mai 2026
Zuletzt verifiziert
1. Mai 2026
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Zusätzliche relevante MeSH-Bedingungen
Andere Studien-ID-Nummern
- CLIN-60240-032
Plan für individuelle Teilnehmerdaten (IPD)
Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?
JA
Beschreibung des IPD-Plans
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-Sharing-Zeitrahmen
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-Sharing-Zugriffskriterien
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
Arzneimittel- und Geräteinformationen, Studienunterlagen
Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt
Nein
Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
Nein
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