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

June 2, 2026 updated by: CSL Behring

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Austria
      • Linz, Austria, 4021
        • Recruiting
        • Kepler Universitätsklinikum - Med Campus III.
  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V6Z 2K5
        • Recruiting
        • Providence Health Care Society
  • Germany
    • Baden-Wurttemberg
      • Ulm, Baden-Wurttemberg, Germany, 89081
        • Recruiting
        • Universitaetsklinikum Ulm
    • Hesse
      • Frankfurt am Main, Hesse, Germany, 60596
        • Active, not recruiting
        • HZRM Haemophilie-Zentrum Rhein Main
    • Lower Saxony
      • Hanover, Lower Saxony, Germany, 30625
        • Active, not recruiting
        • Medizinische Hochschule Hannover
    • Saxony
      • Leipzig, Saxony, Germany, 01307
        • Recruiting
        • Universitaetsklinikum Carl Gustav Carus TU Dresden
    • Schleswig-Holstein
      • Hamburg, Schleswig-Holstein, Germany, 23538
        • Recruiting
        • Universitaetsklinikum Schleswig Holstein - Campus Luebeck
  • United Kingdom
    • Cambridgeshire
      • Cambridge, Cambridgeshire, United Kingdom, CB2 0QQ
        • Recruiting
        • Addenbrooke's Hospital
    • Greater London
      • London, Greater London, United Kingdom, E1 1RD
        • Recruiting
        • Barts Health NHS Trust
    • Greater Manchester
      • Manchester, Greater Manchester, United Kingdom, M13 9WL
        • Recruiting
        • Manchester Royal Infirmary
    • South Glamorgan
      • Cardiff, South Glamorgan, United Kingdom, CF14 4XU
        • Recruiting
        • Cardiff & Vale University HB
    • Surrey
      • Frimley, Surrey, United Kingdom, GU16 7UJ
        • Recruiting
        • Frimley Park Hospital
    • West Midlands
      • Birmingham, West Midlands, United Kingdom, B 9 5SS
        • Recruiting
        • Birmingham Heartlands Hospital
    • West Yorkshire
      • Leeds, West Yorkshire, United Kingdom, LS9 7TF
        • Recruiting
        • Leeds Teaching Hospitals NHS Trust
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85248
        • Recruiting
        • Medical Research of Arizona
    • Florida
      • Miami, Florida, United States, 33183
        • Recruiting
        • Sirven and Associates
    • Illinois
      • Saint Charles, Illinois, United States, 60175-4827
        • Recruiting
        • Asthma and Allergy Wellness Center
    • Kentucky
      • Louisville, Kentucky, United States, 40217
        • Not yet recruiting
        • Family Allergy and Asthma
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Active, not recruiting
        • John Hopkins University
    • Missouri
      • St Louis, Missouri, United States, 63141
        • Active, not recruiting
        • Washington University
    • New Jersey
      • Belleville, New Jersey, United States, 07109
        • Recruiting
        • Riverside Medical Group, Belleville

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participants with the diagnosis of HAE and who will initiate garadacimab treatment per routine clinical practice will be enrolled.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Garadacimab
This cohort will include all participants diagnosed with HAE and who will be prescribed garadacimab treatment as per routine clinical practice.
Other: No intervention
No intervention will be administered as part of this study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
Up to 24 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants with Adverse Events (AEs), Severity of AEs, Serious AEs (SAEs), and Treatment-related AEs
Time Frame: Up to 48 months
Up to 48 months
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.
Baseline to 48 months
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.
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
Up to Month 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CSL Behring

Investigators

  • Study Director: Global Program Director, CSL Behring

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 21, 2025

Primary Completion (Estimated)

August 31, 2028

Study Completion (Estimated)

August 31, 2030

Study Registration Dates

First Submitted

May 23, 2025

First Submitted That Met QC Criteria

May 23, 2025

First Posted (Actual)

June 3, 2025

Study Record Updates

Last Update Posted (Actual)

June 3, 2026

Last Update Submitted That Met QC Criteria

June 2, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CSL312_4001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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