Progressive Familial Intrahepatic CholestasisNCT07185919
A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea
An observational study of Progressive Familial Intrahepatic Cholestasis, run by Ipsen at 5 sites in South Korea.
Trial summary
This study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) as they use odevixibat in their daily lives. Odevixibat is a medicine that helps people with PFIC, a type of rare disease that makes their liver not work well and causes itching and yellow skin. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Progressive Familial Intrahepatic Cholestasis
- How many people
- 10 participants
- Where
- 5 sitesSouth Korea
- Study dates
- 30 Oct 2025to 30 Sep 2032
- How long will I take part?
- 7 Years
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The registry population will comprise participants with PFIC (all types) enrolled into the Ipsen odevixibat PFIC registry. Participants with PFIC who have been prescribed odevixibat by their treating physician will be eligible.
Who may be able to join
- Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enrol the participant in this registry) by their treating physician
- On (or starting) active odevixibat treatment Note: Participants can remain in the registry during odevixibat treatment interruptions
Who cannot join
- 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 South Korea.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
South Korea5 sites, 2 recruiting
- Recruiting
Pusan National University Yangsan Hospital
Gyeongsang
- Active, not recruiting
Asan Medical Center (AMC)
Seoul
- Active, not recruiting
Samsung Medical Center
Seoul
- Active, not recruiting
Seoul National University Hospital
Seoul
- Recruiting
Severance Hospital, Yonsei University Health System
Seoul
About this study
Official title: Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay)
This study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) as they use odevixibat in their daily lives. Odevixibat is a medicine that helps people with PFIC, a type of rare disease that makes their liver not work well and causes itching and yellow skin. 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. Obevixibat was approved by the Ministry of Food and Drug Safety (MFDS) in South Korea on 23 August 2024.
This study will collect information to see how well and how safe odevixibat is in the long run for participants in South Korea.
- Medical subject headings
- Cholestasis, progressive familial intrahepatic 1
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of participants experiencing Adverse Events (AEs)
An Adverse event (AE) is any untoward medical occurrence, temporally associated with the use of study intervention, whether or not related to the study intervention.
Measured: From first ICF signature and up to end of data collection (approximately 7 years of data collection)
Secondary outcomes (4)
Event-free survival (EFS)
EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death
Measured: From first ICF signature and up to end of data collection (approximately 7 years of data collection)
Surgical biliary diversion-free survival
Surgical biliary diversion-free survival is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death
Measured: From first ICF signature and up to end of data collection (approximately 7 years of data collection)
Liver transplant-free survival
Liver transplant-free survival is defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death
Measured: From first ICF signature and up to end of data collection (approximately 7 years of data collection)
Overall survival (OS)
OS is defined as the time from the start of odevixibat treatment to death
Measured: From first ICF signature and up to end of data collection (approximately 7 years of data collection)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2025
- Study start30 Oct 2025
- Primary completion30 Sep 2032 (estimated)
- Study completion30 Sep 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Progressive Familial Intrahepatic CholestasisStudies of the same condition, open and under way.
- More trials run by IpsenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 10 participants (estimated)
- Target duration
- 7 Years
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07185919
- Sponsor's protocol ID
- CLIN-60240-030
- Status last verified
- Oct 2026
- First submitted
- 15 Sep 2025
- Last updated
- 23 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.