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

Study identification

• EU PAS number:
  EUPAS1000000679
• Study ID:
  1000000679
• 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:
  Korea, Republic of
• Study description:
  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.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Ipsen Pharma

Study timelines

Date when funding contract was signed

Actual:25/09/2024

Study start date

Planned:30/09/2025

Actual:30/10/2025

Date of final study report

Planned:31/01/2032

Sources of funding

Pharmaceutical company and other private sector 

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

NCT07185919

Link to ClinicalTrials.gov