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

NCT ID: NCT07001280

Last Updated: 2025-12-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-07-21

Study Completion Date

2030-08-31

Brief Summary

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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.

Detailed Description

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Conditions

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Hereditary Angioedemas

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Garadacimab

This cohort will include all participants diagnosed with HAE and who will be prescribed garadacimab treatment as per routine clinical practice.

No intervention

Intervention Type OTHER

No intervention will be administered as part of this study.

Interventions

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No intervention

No intervention will be administered as part of this study.

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Program Director

Role: STUDY_DIRECTOR

CSL Behring

Locations

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Medical Research of Arizona

Phoenix, Arizona, United States

Site Status RECRUITING

John Hopkins University

Baltimore, Maryland, United States

Site Status ACTIVE_NOT_RECRUITING

Washington University

St Louis, Missouri, United States

Site Status ACTIVE_NOT_RECRUITING

Riverside Medical Group, Belleville

Belleville, New Jersey, United States

Site Status RECRUITING

HZRM Haemophilie-Zentrum Rhein Main

Frankfurt am Main, Hesse, Germany

Site Status ACTIVE_NOT_RECRUITING

Medizinische Hochschule Hannover

Hanover, Lower Saxony, Germany

Site Status ACTIVE_NOT_RECRUITING

Universitaetsklinikum Carl Gustav Carus TU Dresden

Leipzig, Saxony, Germany

Site Status RECRUITING

Universitaetsklinikum Schleswig Holstein - Campus Luebeck

Hamburg, Schleswig-Holstein, Germany

Site Status RECRUITING

Charité - Campus Charité Mitte

Berlin, , Germany

Site Status RECRUITING

Countries

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United States Germany

Central Contacts

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Trial Registration Coordinator

Role: CONTACT

+1 610-878-4697

Facility Contacts

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Charite

Role: primary

Other Identifiers

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CSL312_4001

Identifier Type: -

Identifier Source: org_study_id

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