Progressive Familial Intrahepatic CholestasisNCT07588880
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
An observational study of Progressive Familial Intrahepatic Cholestasis, run by Ipsen at 6 sites in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Progressive Familial Intrahepatic Cholestasis
- How many people
- 20 participants
- Where
- 6 sitesChina
- Study dates
- 16 Apr 2026to 30 Apr 2031
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- 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
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 China
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
China6 sites, 2 recruiting
- Active, not recruiting
Capital Children's Medical Center
Beijing
- Active, not recruiting
Chongqing Medical University - Children's Hospital
Chongqing
- Recruiting
Hunan Children's Hospital
Hunan
- Active, not recruiting
Nanjing Medical University-Nanjing Children's Hospital
Nanjing
- Recruiting
Children's Hospital of Fudan University Endocrinology and Metabolism
Shanghai
- Active, not recruiting
Xiamen Children's Hospital
Xiamen
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 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).
Read more
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.
- 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 AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment
Measured: 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)
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
Measured: 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.
Secondary outcomes (6)
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 5 years of data collection
Surgical biliary diversion-free survival
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 5 years of data collection)
Liver transplant-free survival
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 5 years of data collection)
Overall survival
Defined as time from the start of odevixibat treatment to death.
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Pruritus improvement
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.
Measured: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in serum bile acid
Change from baseline assessed by measuring serum bile acid levels at each patient visit.
Measured: From baseline and up to end of data collection (approximately 5 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 posted15 May 2026
- Study start16 Apr 2026
- Primary completion30 Apr 2031 (estimated)
- Study completion30 Apr 2031 (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
- 20 participants (estimated)
- Target duration
- 5 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
- NCT07588880
- Sponsor's protocol ID
- CLIN-60240-032
- Status last verified
- Oct 2026
- First submitted
- 8 May 2026
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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).
- ClinicalTrials.gov ID
- NCT07588880
- Acronym / sponsor ID
- CLIN-60240-032
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- 16 Apr 2026 (actual)
- Primary completion
- 30 Apr 2031 (estimated)
- Study completion
- 30 Apr 2031 (estimated)
- First posted
- 15 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Target follow-up
- 5 Years
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 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.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Percentage of participants experiencing adverse events (AEs)
Time frame: 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
Percentage of participants experiencing serious adverse events (SAEs)
Time frame: 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
6 secondary outcomes
Event-free survival (EFS)
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection
Surgical biliary diversion-free survival
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Liver transplant-free survival
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Overall survival
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Pruritus improvement
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in serum bile acid
Time frame: From baseline and up to end of data collection (approximately 5 years of data collection
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2026.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ipsen Medical Director study director
Ipsen
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Progressive Familial Intrahepatic Cholestasis, run by Ipsen at 6 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ipsen Clinical Study Enquiries (See e-mail, Clinical.trials@ipsen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of participants experiencing adverse events (AEs) (time frame: 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.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2026.