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 augustus 2026 bijgewerkt door: 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.
Studie Overzicht
Toestand
Werving
Studietype
Observationeel
Inschrijving (Geschat)
20
Contacten en locaties
In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.
Studiecontact
- Naam: Ipsen Clinical Study Enquiries
- Telefoonnummer: See e-mail
- E-mail: clinical.trials@ipsen.com
Studie Locaties
-
-
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Beijing, China
- Actief, niet wervend
- Capital Children's Medical Center
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Chongqing, China
- Actief, niet wervend
- Chongqing Medical University - Children's Hospital
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Hunan, China
- Werving
- Hunan Children's Hospital
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Nanjing, China
- Actief, niet wervend
- Nanjing Medical University-Nanjing Children's Hospital
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Shanghai, China
- Werving
- Children's Hospital of Fudan University Endocrinology and Metabolism
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Xiamen, China
- Actief, niet wervend
- Xiamen Children's Hospital
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-
Deelname Criteria
Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Kind
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Nee
Bemonsteringsmethode
Niet-waarschijnlijkheidssteekproef
Studie Bevolking
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.
Beschrijving
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
Studie plan
Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.
Hoe is de studie opgezet?
Ontwerpdetails
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
Percentage of participants experiencing adverse events (AEs)
Tijdsspanne: 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)
Tijdsspanne: 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
|
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.
|
Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
Event-free survival (EFS)
Tijdsspanne: 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
|
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Surgical biliary diversion-free survival
Tijdsspanne: 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.
|
From first ICF signature and up to end of data collection (approximately 5 years of data collection)
|
|
Liver transplant-free survival
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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)
|
|
Change from baseline in serum bile acid
Tijdsspanne: 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.
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From baseline and up to end of data collection (approximately 5 years of data collection
|
Medewerkers en onderzoekers
Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.
Sponsor
Onderzoekers
- Studie directeur: Ipsen Medical Director, Ipsen
Studie record data
Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.
Bestudeer belangrijke data
Studie start (Werkelijk)
16 april 2026
Primaire voltooiing (Geschat)
30 april 2031
Studie voltooiing (Geschat)
30 april 2031
Studieregistratiedata
Eerst ingediend
8 mei 2026
Eerst ingediend dat voldeed aan de QC-criteria
14 mei 2026
Eerst geplaatst (Werkelijk)
15 mei 2026
Updates van studierecords
Laatste update geplaatst (Werkelijk)
31 augustus 2026
Laatste update ingediend die voldeed aan QC-criteria
28 augustus 2026
Laatst geverifieerd
1 augustus 2026
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- CLIN-60240-032
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
JA
Beschrijving IPD-plan
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-tijdsbestek voor delen
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-toegangscriteria voor delen
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Nee
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
Nee
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