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

NCT07001280 | Recruiting

• 1 other identifier: CSL312_4001
• Study Type: observational
• Target: 200
• Locations: 3 countries
• Sites: 18

Brief 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. 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

Keywords

Angioedema; Genetic disorder; Long term prophylaxis; Health-related quality of life

Outcome Measures

Primary Outcomes (1)

• 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).

  Up to 24 months

Secondary Outcomes (4)

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

  Up to 48 months

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

  Baseline to 48 months

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

  Baseline to 48 months

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

  Up to Month 24

Study Arms (1)

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

Interventions

No intervention OTHER

No intervention will be administered as part of this study.

Garadacimab

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• \. Participants aged greater than or equal to (\>=) 12 years at enrollment.
• \. Participants with clinical and/or laboratory confirmed diagnosis of HAE.
• \. 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.
• \. 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).
• \. Ability to use an electronic device such as a smartphone or a computer for data collection in the study.

You may not qualify if:

• \. Participants with a concomitant diagnosis of another form of angioedema such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria (histaminergic angioedema).
• \. 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.

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (18)

Medical Research of Arizona

Phoenix, Arizona, 85248, United States

RECRUITING

Asthma and Allergy Wellness Center

Saint Charles, Illinois, 60175-4827, United States

RECRUITING

John Hopkins University

Baltimore, Maryland, 21287, United States

ACTIVE NOT RECRUITING

Washington University

St Louis, Missouri, 63141, United States

ACTIVE NOT RECRUITING

Riverside Medical Group, Belleville

Belleville, New Jersey, 07109, United States

RECRUITING

Universitaetsklinikum Ulm

Ulm, Baden-Wurttemberg, 89081, Germany

RECRUITING

HZRM Haemophilie-Zentrum Rhein Main

Frankfurt am Main, Hesse, 60596, Germany

ACTIVE NOT RECRUITING

Medizinische Hochschule Hannover

Hanover, Lower Saxony, 30625, Germany

ACTIVE NOT RECRUITING

Universitaetsklinikum Carl Gustav Carus TU Dresden

Leipzig, Saxony, 01307, Germany

RECRUITING

Universitaetsklinikum Schleswig Holstein - Campus Luebeck

Hamburg, Schleswig-Holstein, 23538, Germany

RECRUITING

Charité - Campus Charité Mitte

Berlin, 10117, Germany

RECRUITING

Addenbrooke's Hospital

Cambridge, Cambridgeshire, CB2 0QQ, United Kingdom

RECRUITING

Barts Health NHS Trust

London, Greater London, E1 1RD, United Kingdom

RECRUITING

Manchester Royal Infirmary

Manchester, Greater Manchester, M13 9WL, United Kingdom

RECRUITING

Cardiff & Vale University HB

Cardiff, South Glamorgan, CF14 4XU, United Kingdom

RECRUITING

Frimley Park Hospital

Frimley, Surrey, GU16 7UJ, United Kingdom

RECRUITING

Birmingham Heartlands Hospital

Birmingham, West Midlands, B 9 5SS, United Kingdom

RECRUITING

Leeds Teaching Hospitals NHS Trust

Leeds, West Yorkshire, LS9 7TF, United Kingdom

RECRUITING

MeSH Terms

Conditions

Angioedemas, Hereditary; Angioedema; Genetic Diseases, Inborn

Condition Hierarchy (Ancestors)

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

Study Officials

• Global Program Director

  CSL Behring

  STUDY DIRECTOR

Central Study Contacts

Trial Registration Coordinator

CONTACT

+1 610-878-4697; clinicaltrials@cslbehring.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 23, 2025
• First Posted: June 3, 2025
• Study Start: July 21, 2025
• Primary Completion (Estimated): August 31, 2028
• Study Completion (Estimated): August 31, 2030
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (5); DE (6); GB (7)