Garadacimab to prevent hereditary angioedema attacks
Garadacimab Real-world Treatment Outcomes of Effectiveness, Safety, and Quality-of-Life in Patients With HAE (GREAT Study)
This study will test whether garadacimab is safe and reduces attack frequency for people aged 12 and older with hereditary angioedema who are starting the medication in routine care.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 200 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | CSL Behring Industry-sponsored |
| Drugs / interventions | garadacimab |
| Locations | 18 sites (Phoenix, Arizona and 17 other locations) |
| Trial ID | NCT07001280 on ClinicalTrials.gov |
What this trial studies
This is a multinational, multicenter prospective observational cohort following patients newly initiating garadacimab in routine clinical practice. Each participant will be followed for 48 months after their first dose, with data collected from an electronic HAE eDiary, medical records, and routine clinic visits. The study extracts 12 months of retrospective healthcare resource and safety data from records and 3 months of retrospective attack data from the eDiary. No treatments are assigned by the study; garadacimab use is determined by the treating physician per local labeling.
Who should consider this trial
Good fit: People aged 12 years or older with a confirmed diagnosis of hereditary angioedema who are newly starting garadacimab as prescribed by their physician, can provide consent (or have guardian consent), and can use a smartphone or computer for data entry are ideal candidates.
Not a fit: Patients with other forms of angioedema (such as acquired, idiopathic, or histaminergic) or those not starting garadacimab are not eligible and are unlikely to receive benefit from participation.
Why it matters
Potential benefit: If successful, this could show that garadacimab reduces attack frequency and improves quality of life for people with HAE in everyday clinical practice.
How similar studies have performed: Previous clinical trials of garadacimab have reported promising reductions in HAE attack rates, but long-term real-world safety and effectiveness data are still limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 1\. Participants aged greater than or equal to (\>=) 12 years at enrollment. * 2\. Participants with clinical and/or laboratory confirmed diagnosis of HAE. * 3\. Participants newly initiating garadacimab, as prescribed according to the decision of the treating physician per routine clinical practice and in accordance with the indication per the approved local label, independent of and prior to enrollment in the study. * 4\. Willing and able to provide written informed consent and/or assent by parent or legal guardian for children less than (\<) 18 years of age (or legal age of consent in the respective countries). * 5\. Ability to use an electronic device such as a smartphone or a computer for data collection in the study. Exclusion Criteria: * 1\. Participants with a concomitant diagnosis of another form of angioedema such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria (histaminergic angioedema). * 2\. Participants participating in any ongoing interventional clinical study, including interventional studies with garadacimab. Participants in this study who later chose to enroll in any interventional clinical study (including garadacimab) will be discontinued from this study.
Where this trial is running
Phoenix, Arizona and 17 other locations
- Medical Research of Arizona — Phoenix, Arizona, United States (Recruiting)
- Asthma and Allergy Wellness Center — Saint Charles, Illinois, United States (Recruiting)
- John Hopkins University — Baltimore, Maryland, United States (Active_not_recruiting)
- Washington University — St Louis, Missouri, United States (Active_not_recruiting)
- Riverside Medical Group, Belleville — Belleville, New Jersey, United States (Recruiting)
- Universitaetsklinikum Ulm — Ulm, Baden-Wurttemberg, Germany (Recruiting)
- HZRM Haemophilie-Zentrum Rhein Main — Frankfurt am Main, Hesse, Germany (Active_not_recruiting)
- Medizinische Hochschule Hannover — Hanover, Lower Saxony, Germany (Active_not_recruiting)
- Universitaetsklinikum Carl Gustav Carus TU Dresden — Leipzig, Saxony, Germany (Recruiting)
- Universitaetsklinikum Schleswig Holstein - Campus Luebeck — Hamburg, Schleswig-Holstein, Germany (Recruiting)
- Charité - Campus Charité Mitte — Berlin, Germany (Recruiting)
- Addenbrooke's Hospital — Cambridge, Cambridgeshire, United Kingdom (Recruiting)
- Barts Health NHS Trust — London, Greater London, United Kingdom (Recruiting)
- Manchester Royal Infirmary — Manchester, Greater Manchester, United Kingdom (Recruiting)
- Cardiff & Vale University HB — Cardiff, South Glamorgan, United Kingdom (Recruiting)
- Frimley Park Hospital — Frimley, Surrey, United Kingdom (Recruiting)
- Birmingham Heartlands Hospital — Birmingham, West Midlands, United Kingdom (Recruiting)
- Leeds Teaching Hospitals NHS Trust — Leeds, West Yorkshire, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Trial Registration Coordinator
- Email: clinicaltrials@cslbehring.com
- Phone: +1 610-878-4697
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.