An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients with Hemophilia B (CSL222_4001)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/106067
• EU PAS number:
  EUPAS106066
• Study ID:
  106067
• Official title and acronym:
  An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients with Hemophilia B (CSL222_4001)
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Belgium

  Denmark

  Finland

  France

  Germany

  Ireland

  Italy

  Netherlands

  Norway

  Spain

  Sweden

  Switzerland

  United Kingdom

  United States
• Study description:
  This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.
• Study status:
  Planned

Research institutions and networks

Institutions

CSL Behring

Study timelines

Date when funding contract was signed

Planned: 15/08/2023

Study start date

Planned: 15/09/2023

Data analysis start date

Planned: 24/08/2043

Date of final study report

Planned: 15/01/2044

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

CSL Behring

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 1 (imposed as condition of marketing authorisation)