Hereditary angioedema Hereditary angioedema
Conditions
Interventions
No investigational product.
Sponsors
CSL Behring LLC
Eligibility
Age
12 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Patients aged =12 years at enrollment. 2. Patients with clinical and/or laboratory confirmed diagnosis of HAE. 3. Patients 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 <18 years of age (or legal age of consent in the respective countries). Note: The choice of the terms of parental consent or parental permission in different regions may reflect local legal / regulatory and ethical considerations. 5. Ability to use an electronic device such as a smartphone or a computer for data collection in the study.
Exclusion criteria
Exclusion criteria: 1. Patients with a concomitant diagnosis of another form of angioedema such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria (histaminergic angioedema). 2. Patients participating in any ongoing interventional clinical study, including interventional studies with garadacimab. Patients in this study who later chose to enroll in any interventional clinical study (including garadacimab) will be discontinued from this study. Note: If the patient is currently enrolled in other observational studies related to garadacimab, the patient may be enrolled in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| p.32 of the protocol:Primary Objective: Treatment Effectiveness • 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 Outcome:• Incidence rate ratio (IRR) of HAE attacks while on treatment with garadacimab compared to patient-reported history of HAE attacks during 3 months prior to enrollment | — |
Secondary
| Measure | Time frame |
|---|---|
| • To describe the real-world short and long-term safety profile of garadacimab in adult and adolescent (ages 12-17 years) patients with HAE over 48 months of follow-up • To assess different aspects of HRQoL, as reported by patients, with HAE during the course of their treatment with garadacimab, as well as relative to HRQoL prior to treatment with garadacimab over 48 months of follow-up • To describe the association of garadacimab treatment with the onset of attack prevention, as well as the durability of disease control over 24 months of follow-up • To describe adherence / compliance to garadacimab treatment over 48 months of follow-up • 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, with a focus on HAE attacks using on-demand therapy (ODT) and HAE attacks by severity (mild, moderate, and severe)• To describe characteristics of HAE attacks while on treatment with garadacimab over 48 months of follow-up • To assess patients’ satisfaction with their HAE treatment before and after initiation of garadacimab treatment over 48 months of follow-up • To assess the association between effectiveness, HRQoL, and treatment satisfaction over 24 months of follow-up • To describe garadacimab exposure and treatment patterns during garadacimab treatment (eg, use of ODT to manage attacks while on treatment, treatment interruptions, switches to other HAE treatments following garadacimab discontinuation) over 48 months of follow-up • To describe treatment patterns before initiating garadacimab (eg, treatment-naïve, ODT, short-term prophylaxis [STP], LTP), including the duration of treatment and any observed patterns of switching treatments • To determine the frequency of healthcare visits (ie, physician visits, emergency room (ER) / urgent care visits, inpatient stays, referrals, surgeries, and procedu | — |
Countries
Austria, Canada, France, Germany, Hungary, Italy, Japan, Netherlands, United Kingdom, United States
Contacts
Public ContactCH Nenci
CSL Innovation GmbH
Outcome results
None listed