Non-Interventional Post-Authorisation Study to Evaluate the Safety, Tolerability and Effectiveness of Berotralstat for Patients with Hereditary Angioedema (HAE) in a Real-World Setting (APeX-N)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/43576
• EU PAS number:
  EUPAS43575
• Study ID:
  43576
• Official title and acronym:
  Non-Interventional Post-Authorisation Study to Evaluate the Safety, Tolerability and Effectiveness of Berotralstat for Patients with Hereditary Angioedema (HAE) in a Real-World Setting (APeX-N)
• DARWIN EU® study:
  No
• Study countries:
  France

  Germany

  Norway

  Sweden

  United Kingdom
• Study description:
  The purpose of this study is to evaluate the long-term safety and effectiveness of berotralstat in a voluntary category 3 PASS study in a real-world setting in accordance with EMA- and MHRA-approved labelling.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Royal Free Hospital

Study timelines

Date when funding contract was signed

Planned: 03/05/2021

Actual: 27/05/2022

Study start date

Planned: 03/01/2022

Actual: 26/07/2022

Date of final study report

Planned: 05/04/2027

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

BioCryst Pharmaceuticals

Study protocol

Updated protocol

BCX7353-401 Protocol v3.0_Redacted_10 Jul 2024.pdf

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)