Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who take Odevixibat (Bylvay)

Study summary

No information provided.

Study identification

• EU PAS number:
  EUPAS1000000975
• Study ID:
  1000000975
• Official title and acronym:
  Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who take Odevixibat (Bylvay)
• DARWIN EU® study:
  No
• Study countries:
  China
• Study description:
  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 status:
  Ongoing

Study timelines

Date when funding contract was signed

Planned:07/10/2025

Actual:07/10/2025

Study start date

Planned:16/04/2026

Actual:16/04/2026

Date of final study report

Planned:30/04/2031

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Ipsen Pharma

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable

Other study registration identification numbers and links

CLIN‑60240‑032