Hereditary AngioedemasGREATNCT07001280
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
An observational study of Hereditary Angioedemas, run by CSL Behring at 22 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedemas
- How many people
- 200 participants
- Where
- 22 sitesin 5 countries
- Study dates
- 21 Jul 2025to 31 Aug 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants with the diagnosis of HAE and who will initiate garadacimab treatment per routine clinical practice will be enrolled.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
22 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
22 sites in 5 countries
United States7 sites, 5 recruiting
- Active, not recruiting
John Hopkins University
Baltimore, Maryland, 21287
- Recruiting
Riverside Medical Group, Belleville
Belleville, New Jersey, 07109
- Not yet recruiting
Family Allergy and Asthma
Louisville, Kentucky, 40217
- Recruiting
Sirven and Associates
Miami, Florida, 33183
- Recruiting
Medical Research of Arizona
Phoenix, Arizona, 85248
- Recruiting
Asthma and Allergy Wellness Center
Saint Charles, Illinois, 60175-4827
- Active, not recruiting
Washington University
St Louis, Missouri, 63141
United Kingdom7 sites, 7 recruiting
- Recruiting
Birmingham Heartlands Hospital
Birmingham, West Midlands, B 9 5SS
- Recruiting
Addenbrooke's Hospital
Cambridge, Cambridgeshire, CB2 0QQ
- Recruiting
Cardiff & Vale University HB
Cardiff, South Glamorgan, CF14 4XU
- Recruiting
Frimley Park Hospital
Frimley, Surrey, GU16 7UJ
- Recruiting
Leeds Teaching Hospitals NHS Trust
Leeds, West Yorkshire, LS9 7TF
- Recruiting
Barts Health NHS Trust
London, Greater London, E1 1RD
- Recruiting
Manchester Royal Infirmary
Manchester, Greater Manchester, M13 9WL
Germany6 sites, 4 recruiting
- Recruiting
Charité - Campus Charité Mitte
Berlin, 10117
Charite, clinicaltrials@cslbehring.com
- Active, not recruiting
HZRM Haemophilie-Zentrum Rhein Main
Frankfurt am Main, Hesse, 60596
- Recruiting
Universitaetsklinikum Schleswig Holstein - Campus Luebeck
Hamburg, Schleswig-Holstein, 23538
- Active, not recruiting
Medizinische Hochschule Hannover
Hanover, Lower Saxony, 30625
- Recruiting
Universitaetsklinikum Carl Gustav Carus TU Dresden
Leipzig, Saxony, 01307
- Recruiting
Universitaetsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081
Austria1 site, 1 recruiting
- Recruiting
Kepler Universitätsklinikum - Med Campus III.
Linz, 4021
Canada1 site, 1 recruiting
- Recruiting
Providence Health Care Society
Vancouver, British Columbia, V6Z 2K5
About this study
Official title: 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.
- Conditions
- Hereditary Angioedemas
- Being tested
- No intervention
- Keywords
- Angioedema, Genetic disorder, Long term prophylaxis, Health-related quality of life
- Medical subject headings
- Angioedemas, Hereditary, Angioedema, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
No intervention will be administered as part of this study.
| Study group | Role | What they receive |
|---|---|---|
| Garadacimab | This cohort will include all participants diagnosed with HAE and who will be prescribed garadacimab treatment as per routine clinical practice. Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence Rate Ratio (IRR) of HAE Attacks
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).
Measured: Up to 24 months
Secondary outcomes (4)
Number of Participants with Adverse Events (AEs), Severity of AEs, Serious AEs (SAEs), and Treatment-related AEs
Measured: Up to 48 months
Change From Baseline in Angioedema Control Test (AECT) Total Score
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.
Measured: Baseline to 48 months
Change From Baseline in Angioedema Quality of Life Questionnaire (AE-QoL) Total Score
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.
Measured: Baseline to 48 months
Percentage of Participants With Reduction in Monthly HAE Attacks and who Achieved HAE Attack-free Status
Measured: Up to Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2025
- Study start21 Jul 2025
- Primary completion31 Aug 2028 (estimated)
- Study completion31 Aug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary AngioedemasStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Program Director, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07001280
- Sponsor's protocol ID
- CSL312_4001
- Acronym
- GREAT
- Status last verified
- May 2026
- First submitted
- 23 May 2025
- Last updated
- 3 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Garadacimab Real-world Treatment Outcomes of Effectiveness, Safety, and Quality-of-Life in Patients With HAE (GREAT Study)
- ClinicalTrials.gov ID
- NCT07001280
- Acronym / sponsor ID
- GREAT / CSL312_4001
- Conditions
- Hereditary Angioedemas
- Keywords
- Angioedema, Genetic disorder, Long term prophylaxis, Health-related quality of life
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 21 Jul 2025 (actual)
- Primary completion
- 31 Aug 2028 (estimated)
- Study completion
- 31 Aug 2030 (estimated)
- First posted
- 3 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Garadacimab | – | Other: No interventionThis cohort will include all participants diagnosed with HAE and who will be prescribed garadacimab treatment as per routine clinical practice. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with the diagnosis of HAE and who will initiate garadacimab treatment per routine clinical practice will be enrolled.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionNo intervention will be administered as part of this study. | Other | Garadacimab |
Primary outcome
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).
4 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Global Program Director study director
CSL Behring
22 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Angioedemas, run by CSL Behring at 22 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, United Kingdom, Germany, Austria, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Registration Coordinator (+1 610-878-4697, clinicaltrials@cslbehring.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence Rate Ratio (IRR) of HAE Attacks (time frame: Up to 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2026.