Long-term outcomes of odevixibat (Bylvay) for people with PFIC in South Korea
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 will see how well odevixibat controls symptoms and how safe it is over time for people with Progressive Familial Intrahepatic Cholestasis (PFIC) in South Korea.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 10 (estimated) |
| Sex | All |
| Sponsor | Ipsen Industry-sponsored |
| Locations | 3 sites (Gyeongsang and 2 other locations) |
| Trial ID | NCT07185919 on ClinicalTrials.gov |
What this trial studies
This is a multicenter observational registry collecting real-world information from people with any type of PFIC who have been prescribed odevixibat as part of routine care. Data will include safety events, symptom changes (including itching), liver-related outcomes, and long-term treatment patterns, with participants followed while on treatment and during permitted interruptions. Patients already enrolled in interventional odevixibat trials or other PFIC interventional trials are excluded. The registry is conducted at major tertiary centers in South Korea and will capture outcomes under usual clinical practice rather than a controlled protocol.
Who should consider this trial
Good fit: People in South Korea with a diagnosis of any type of PFIC who have been prescribed odevixibat by their treating physician and are willing to share routine health data are ideal candidates.
Not a fit: People with contraindications to odevixibat, those currently participating in an interventional odevixibat or other PFIC interventional trial, or those not taking odevixibat are unlikely to benefit from this registry.
Why it matters
Potential benefit: If successful, the registry could provide long-term, real-world evidence about safety and sustained symptom benefit from odevixibat that helps patients and doctors make treatment decisions.
How similar studies have performed: Randomized trials supported regulatory approvals showing odevixibat can reduce itching in infants with PFIC, but long-term real-world outcome data are limited, so this registry builds on prior trial results.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion criteria: 1. 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 2. On (or starting) active odevixibat treatment Note: Participants can remain in the registry during odevixibat treatment interruptions Exclusion criteria: 1. Currently participating in a clinical trial with odevixibat 2. Currently participating in any interventional clinical trial for PFIC 3. Have any contraindication to odevixibat as per the approved label in South Korea.
Where this trial is running
Gyeongsang and 2 other locations
- Pusan National University Yangsan Hospital — Gyeongsang, South Korea (Active_not_recruiting)
- Seoul National University Hospital — Seoul, South Korea (Not_yet_recruiting)
- Severance Hospital, Yonsei University Health System — Seoul, South Korea (Recruiting)
Study contacts
- Study coordinator: Ipsen Clinical Study Enquiries
- Email: clinical.trials@ipsen.com
- Phone: See e mail
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.