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This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice. Each participant will be followed for 48 months after index date (date of the first administration of garadacimab).
Patient data will be collected from the HAE eDiary, patient medical records (MRs) and/or during a routine clinical visit and will be entered into the electronic case report form (eCRF) via an electronic data capture (EDC) system. Data pertaining to HAE attacks, prior HAE treatments, retrospective focused safety data collection, and healthcare resource utilization (HCRU) over a look-back period of 12 months prior to the enrollment will be extracted from the MR, and patients will also record retrospective HAE attack related data over a look-back period of 3 months prior to enrollment in the HAE eDiary.
The primary aim of this study is to investigate the real-world effectiveness of garadacimab as measured by HAE attack rate before and after garadacimab initiation in patients with HAE over 24 months of follow-up. The study will aim to complement the data available from the clinical development program on the efficacy, safety, and health-related quality-of-life (HRQoL) in patients with HAE taking garadacimab.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Garadacimab | This cohort will include all participants diagnosed with HAE and who will be prescribed garadacimab treatment as per routine clinical practice. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No intervention | Other | No intervention will be administered as part of this study. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence Rate Ratio (IRR) of HAE Attacks | Monthly HAE attack rate while on treatment with garadacimab, including all HAE attacks, HAE attacks requiring acute treatment (on demand therapy [ODT]), and by severity will be reported. All HAE attacks will be categorized by severity as: Mild (no impact on daily life), Moderate (some impact on daily life), and Severe (severe impact on daily life). | Up to 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Adverse Events (AEs), Severity of AEs, Serious AEs (SAEs), and Treatment-related AEs | Up to 48 months | |
| Change From Baseline in Angioedema Control Test (AECT) Total Score | The AECT includes a set of 4 AECT items attributable to the domains of signs and symptoms, impact and effectiveness of treatment, and the 4th item addresses the important aspect of unpredictability, which correlates strongly with anxiety and fears of recurrent angioedema patients. Score ranges from 0 (minimum) to 16 (maximum). A higher score indicates a higher level of angioedema control. |
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Inclusion Criteria:
Exclusion Criteria:
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Participants with the diagnosis of HAE and who will initiate garadacimab treatment per routine clinical practice will be enrolled.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Trial Registration Coordinator | Contact | +1 610-878-4697 | clinicaltrials@cslbehring.com |
| Name | Affiliation | Role |
|---|---|---|
| Global Program Director | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Medical Research of Arizona | Recruiting | Phoenix | Arizona | 85248 | United States | |
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| Baseline to 48 months |
| Change From Baseline in Angioedema Quality of Life Questionnaire (AE-QoL) Total Score | The AE-QoL includes 17 items across 4 domains: functioning (4 items), fatigue/mood (5 items), fears/shame (6 items), and food (2 items). Total scores range from 0 to 100, where 100 indicates the worst possible impairment of health related QoL. | Baseline to 48 months |
| Percentage of Participants With Reduction in Monthly HAE Attacks and who Achieved HAE Attack-free Status | Up to Month 24 |
| Sirven and Associates |
| Recruiting |
| Miami |
| Florida |
| 33183 |
| United States |
| Asthma and Allergy Wellness Center | Recruiting | Saint Charles | Illinois | 60175-4827 | United States |
| Family Allergy and Asthma | Not yet recruiting | Louisville | Kentucky | 40217 | United States |
| John Hopkins University | Active, not recruiting | Baltimore | Maryland | 21287 | United States |
| Washington University | Active, not recruiting | St Louis | Missouri | 63141 | United States |
| Riverside Medical Group, Belleville | Recruiting | Belleville | New Jersey | 07109 | United States |
| Kepler Universitätsklinikum - Med Campus III. | Recruiting | Linz | 4021 | Austria |
| Providence Health Care Society | Recruiting | Vancouver | British Columbia | V6Z 2K5 | Canada |
| Universitaetsklinikum Ulm | Recruiting | Ulm | Baden-Wurttemberg | 89081 | Germany |
| HZRM Haemophilie-Zentrum Rhein Main | Active, not recruiting | Frankfurt am Main | Hesse | 60596 | Germany |
| Medizinische Hochschule Hannover | Active, not recruiting | Hanover | Lower Saxony | 30625 | Germany |
| Universitaetsklinikum Carl Gustav Carus TU Dresden | Recruiting | Leipzig | Saxony | 01307 | Germany |
| Universitaetsklinikum Schleswig Holstein - Campus Luebeck | Recruiting | Hamburg | Schleswig-Holstein | 23538 | Germany |
| Charité - Campus Charité Mitte | Recruiting | Berlin | 10117 | Germany |
|
| Addenbrooke's Hospital | Recruiting | Cambridge | Cambridgeshire | CB2 0QQ | United Kingdom |
| Barts Health NHS Trust | Recruiting | London | Greater London | E1 1RD | United Kingdom |
| Manchester Royal Infirmary | Recruiting | Manchester | Greater Manchester | M13 9WL | United Kingdom |
| Cardiff & Vale University HB | Recruiting | Cardiff | South Glamorgan | CF14 4XU | United Kingdom |
| Frimley Park Hospital | Recruiting | Frimley | Surrey | GU16 7UJ | United Kingdom |
| Birmingham Heartlands Hospital | Recruiting | Birmingham | West Midlands | B 9 5SS | United Kingdom |
| Leeds Teaching Hospitals NHS Trust | Recruiting | Leeds | West Yorkshire | LS9 7TF | United Kingdom |
| ID | Term |
|---|---|
| D054179 | Angioedemas, Hereditary |
| D000799 | Angioedema |
| D030342 | Genetic Diseases, Inborn |
| ID | Term |
|---|---|
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D000081208 | Hereditary Complement Deficiency Diseases |
| D000081207 | Primary Immunodeficiency Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014581 | Urticaria |
| D017445 | Skin Diseases, Vascular |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D007153 | Immunologic Deficiency Syndromes |
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