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

Официальное название

Garadacimab Real-world Treatment Outcomes of Effectiveness, Safety, and Quality-of-Life in Patients With HAE (GREAT Study)

Обзор

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.

Вмешательства

• Другое No intervention
  No intervention will be administered as part of this study.

Первичные конечные точки

• Incidence Rate Ratio (IRR) of HAE Attacks [Срок оценки: Up to 24 months]

Вторичные конечные точки (4)

• Number of Participants with Adverse Events (AEs), Severity of AEs, Serious AEs (SAEs), and Treatment-related AEs [Срок оценки: Up to 48 months]
• Change From Baseline in Angioedema Control Test (AECT) Total Score [Срок оценки: Baseline to 48 months]
• Change From Baseline in Angioedema Quality of Life Questionnaire (AE-QoL) Total Score [Срок оценки: Baseline to 48 months]
• Percentage of Participants With Reduction in Monthly HAE Attacks and who Achieved HAE Attack-free Status [Срок оценки: Up to Month 24]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 7 центров

• 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

Великобритания · 7 центров

• 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

Германия · 6 центров

• 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

Австрия · 1 центр

• Kepler Universitätsklinikum - Med Campus III. — Linz

Канада · 1 центр

• Providence Health Care Society — Vancouver

Идентификаторы

NCT: NCT07001280 · CSL312_4001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗