A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: No intervention.
- Who it may be relevant to
- Registry conditions: Hereditary Angioedemas. Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Austria, Canada, Germany, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Garadacimab Real-world Treatment Outcomes of Effectiveness, Safety, and Quality-of-Life in Patients With HAE (GREAT Study)
Overview
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.
Interventions
- Other No intervention
No intervention will be administered as part of this study.
Primary outcome measures
- Incidence Rate Ratio (IRR) of HAE Attacks [Time frame: Up to 24 months]
Secondary outcome measures (4)
- Number of Participants with Adverse Events (AEs), Severity of AEs, Serious AEs (SAEs), and Treatment-related AEs [Time frame: Up to 48 months]
- Change From Baseline in Angioedema Control Test (AECT) Total Score [Time frame: Baseline to 48 months]
- Change From Baseline in Angioedema Quality of Life Questionnaire (AE-QoL) Total Score [Time frame: Baseline to 48 months]
- Percentage of Participants With Reduction in Monthly HAE Attacks and who Achieved HAE Attack-free Status [Time frame: Up to Month 24]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 7 centers
- Medical Research of Arizona — Phoenix
- Sirven and Associates — Miami
- Asthma and Allergy Wellness Center — Saint Charles
- Family Allergy and Asthma — Louisville
- John Hopkins University — Baltimore
- Washington University — St Louis
- Riverside Medical Group, Belleville — Belleville
United Kingdom · 7 centers
- Addenbrooke's Hospital — Cambridge
- Barts Health NHS Trust — London
- Manchester Royal Infirmary — Manchester
- Cardiff & Vale University HB — Cardiff
- Frimley Park Hospital — Frimley
- Birmingham Heartlands Hospital — Birmingham
- Leeds Teaching Hospitals NHS Trust — Leeds
Germany · 6 centers
- Universitaetsklinikum Ulm — Ulm
- HZRM Haemophilie-Zentrum Rhein Main — Frankfurt am Main
- Medizinische Hochschule Hannover — Hanover
- Universitaetsklinikum Carl Gustav Carus TU Dresden — Leipzig
- Universitaetsklinikum Schleswig Holstein - Campus Luebeck — Hamburg
- Charité - Campus Charité Mitte — Berlin
Austria · 1 center
- Kepler Universitätsklinikum - Med Campus III. — Linz
Canada · 1 center
- Providence Health Care Society — Vancouver
Identifiers
NCT: NCT07001280 · CSL312_4001