Actively Recruiting
Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay)
Led by Ipsen · Updated on 2026-05-15
20
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are collecting information from people with Progressive Familial Intrahepatic Cholestasis (PFIC), a rare genetic liver disease that affects bile secretion and causes bile acids to build up, leading to symptoms like severe itching. The study focuses on those who take the medicine odevixibat (Bylvay) as part of their regular care in China. Odevixibat is approved for treating itching in PFIC patients aged 6 months and older in China, with prior approvals in Europe and the United States for similar uses. This is a registry-based observational study that tracks participants over about five years. It records long-term safety by monitoring adverse events and serious adverse events. The study also describes how well odevixibat works by looking at outcomes such as survival without surgery, liver transplant-free survival, overall survival, improvement in itching, and changes in bile acid levels. Participants provide informed consent and receive regular follow-up during their treatment. Data collected includes safety events, treatment outcomes, and long-term health status. The study follows participants from the time they sign consent up to five years or 30 days after stopping odevixibat. This approach helps researchers understand the real-world effects and safety of odevixibat in PFIC patients.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with PFIC (all types) and prescribed odevixibat by their treating physician
- Currently on or starting active odevixibat treatment
- Signed informed consent and assent as appropriate
You will not qualify if you...
- 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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to approximately 5 years
Participants who take odevixibat are observed for effectiveness, safety, and long-term outcomes.
Regular visits as per routine clinical care
Trial Site Locations
Total: 1 location
1
Children's Hospital of Fudan University Endocrinology and Metabolism
Shanghai, China
Actively Recruiting
Research Team
I
Ipsen Clinical Study Enquiries
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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