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NCT Number: NCT07001280

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

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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Key information

Conditions

Hereditary Angioedemas Angioedema Angioedemas, Hereditary Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Complement Deficiency Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Immunologic Deficiency Syndromes Primary Immunodeficiency Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Kepler Universitätsklinikum - Med Campus III., Linz, Austria

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

No intervention

Other

No intervention will be administered as part of this study.

Primary outcomes

  1. Incidence Rate Ratio (IRR) of HAE Attacks

    Time frame: 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).

Secondary outcomes

  1. Number of Participants with Adverse Events (AEs), Severity of AEs, Serious AEs (SAEs), and Treatment-related AEs

    Time frame: Up to 48 months

  2. Change From Baseline in Angioedema Control Test (AECT) Total Score

    Time frame: Baseline to 48 months

    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.

  3. Change From Baseline in Angioedema Quality of Life Questionnaire (AE-QoL) Total Score

    Time frame: Baseline to 48 months

    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.

  4. Percentage of Participants With Reduction in Monthly HAE Attacks and who Achieved HAE Attack-free Status

    Time frame: Up to Month 24

Study contacts

Contact information is provided by the study sponsor or research team.

Trial Registration Coordinator

CONTACT

[email protected]

+1 610-878-4697

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

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

Acronym: GREAT

Important dates

Study start
2025
Primary completion
2028
Study completion
2030
First posted
Jun 3, 2025
Registry last updated
Jun 3, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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