Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/1000000441
• EU PAS number:
  EUPAS1000000441
• Study ID:
  1000000441
• Official title and acronym:
  Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)
• DARWIN EU® study:
  No
• Study countries:
  United States
• Study description:
  This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives. Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching. ; The main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with Alagille Syndrome (ALGS) who are receiving ongoing treatment.
• Study status:
  Ongoing

Study timelines

Date when funding contract was signed

Planned: 07/05/2024

Actual: 07/05/2024

Study start date

Planned: 17/03/2025

Actual: 22/04/2025

Date of interim report, if expected

Planned: 30/11/2025

Date of final study report

Planned: 30/05/2030

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Ipsen Pharma

Study protocol

Initial protocol

CLIN-60240-033_16.1.1 Protocol v4.0_Redacted.pdf

Regulatory

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