Retrospective analysis of adolescent patients suffering from hereditary angioedema treated with berotralstat in Europe: patients’ and treatment characteristics

Study summary

No information provided.

Study identification

• EU PAS number:
  EUPAS1000000997
• Study ID:
  1000000997
• Official title and acronym:
  Retrospective analysis of adolescent patients suffering from hereditary angioedema treated with berotralstat in Europe: patients’ and treatment characteristics
• DARWIN EU® study:
  Yes
• Study countries:
  Belgium

  France

  Germany

  Italy

  Spain

  United Kingdom
• Study description:
  This study is an international, retrospective, multicenter observational case series sponsored by BioCryst Ireland Limited. It aims to describe the clinical characteristics, treatment patterns, and outcomes of European adolescent patients (aged 12–17 years) with hereditary angioedema (HAE) treated with berotralstat.; Data will be collected retrospectively from medical records of patients receiving berotralstat as long-term prophylaxis. The study focuses on baseline patient characteristics, disease history, treatment patterns, effectiveness outcomes (e.g., attack frequency and severity), and safety, including adverse events.; Approximately 10 sites across Europe will contribute data on up to 30 patients. The study period runs from Q2 2026 to Q4 2027, with data analysis planned in end of 2027.; The objective is to generate real-world evidence on the use of berotralstat in adolescents, a population underrepresented in clinical trials, to better understand treatment effectiveness, safety, and disease evolution in routine clinical practice.
• Study status:
  Planned

Research institutions and networks

Institutions

Biocryst Ireland Ltd.

Study timelines

Date when funding contract was signed

Planned:10/12/2025

Study start date

Planned:01/09/2026

Date of final study report

Planned:01/01/2028

Sources of funding

Pharmaceutical company and other private sector 

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable