A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)

NCT07001280 · tracked via the Priya Life Science USA tracker

Sponsor

CSL Behring

Phase

Observational

Started

2025-07-21

Last updated

2026-06-03

Condition(s) studied

Hereditary Angioedemas

Investigational drug(s) / intervention(s)

No intervention

No intervention: No intervention will be administered as part of this study.

Study summary

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.

Eligibility

Sex

ALL

Min age

12 Years

Max age

—

Healthy volunteers

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.

Primary outcome measure(s)

Incidence Rate Ratio (IRR) of HAE Attacks — Up to 24 months
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).

Trial sites (22)

Facility	City	Region	Status
Medical Research of Arizona	Phoenix	Arizona	Recruiting
Sirven and Associates	Miami	Florida	Recruiting
Asthma and Allergy Wellness Center	Saint Charles	Illinois	Recruiting
Family Allergy and Asthma	Louisville	Kentucky	Not Yet Recruiting
John Hopkins University	Baltimore	Maryland	Active Not Recruiting
Washington University	St Louis	Missouri	Active Not Recruiting
Riverside Medical Group, Belleville	Belleville	New Jersey	Recruiting
Kepler Universitätsklinikum - Med Campus III.	Linz	Austria	Recruiting
Providence Health Care Society	Vancouver	British Columbia	Recruiting
Universitaetsklinikum Ulm	Ulm	Baden-Wurttemberg	Recruiting
HZRM Haemophilie-Zentrum Rhein Main	Frankfurt am Main	Hesse	Active Not Recruiting
Medizinische Hochschule Hannover	Hanover	Lower Saxony	Active Not Recruiting
Universitaetsklinikum Carl Gustav Carus TU Dresden	Leipzig	Saxony	Recruiting
Universitaetsklinikum Schleswig Holstein - Campus Luebeck	Hamburg	Schleswig-Holstein	Recruiting
Charité - Campus Charité Mitte	Berlin	Germany	Recruiting
Addenbrooke's Hospital	Cambridge	Cambridgeshire	Recruiting
Barts Health NHS Trust	London	Greater London	Recruiting
Manchester Royal Infirmary	Manchester	Greater Manchester	Recruiting
Cardiff & Vale University HB	Cardiff	South Glamorgan	Recruiting
Frimley Park Hospital	Frimley	Surrey	Recruiting
Birmingham Heartlands Hospital	Birmingham	West Midlands	Recruiting
Leeds Teaching Hospitals NHS Trust	Leeds	West Yorkshire	Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07001280 on ClinicalTrials.gov ↗ ← All trials in the USA