GREAT
Research type
Research Study
Full title
Garadacimab Real-world Treatment Outcomes of Effectiveness, Safety, and Quality-of-Life in Patients with HAE
IRAS ID
361392
Contact name
Sinisa Savic
Contact email
Sponsor organisation
CSL Behring GmbH
Clinicaltrials.gov Identifier
NCT07001280, ClinicalTrials.gov
Duration of Study in the UK
4 years, 9 months, 31 days
Research summary
Hereditary Angioedema (HAE) is a rare genetic condition that causes sudden and painful swelling in various parts of the body, including the face, limbs, and airways. These attacks can be unpredictable and, in some cases, life-threatening. Garadacimab is a new medicine designed to prevent HAE attacks before they happen. It works by blocking a protein called activated Factor XII, which plays a role in triggering swelling.
This study will explore how garadacimab works in real-life settings, outside of clinical trials. It will help researchers, doctors, and patients understand how effective and safe the treatment is over time, and how it affects patients’ quality of life.
The study is observational, meaning that researchers will not change or influence the treatment. Instead, they will collect information from patients who are starting garadacimab as part of their usual care. Around 200 patients aged 12 and older will be recruited from outpatient clinics, such as allergy and immunology practices, in several countries including the UK.
Participants will be followed for up to four years. During this time, they will record their symptoms and experiences using a secure electronic diary and complete questionnaires about their health and wellbeing. Information will also be collected from routine medical visits and patient records.
The study will look at how often patients have HAE attacks before and after starting garadacimab, how safe the treatment is, how satisfied patients are with it, and how often they need to visit healthcare services. It will also explore treatment patterns and patient preferences.
This research is funded by CSL Behring, the company that developed garadacimab. The findings may help improve care for people with HAE and guide future treatment decisions.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
25/SC/0337
Date of REC Opinion
20 Nov 2025
REC opinion
Further Information Favourable Opinion