RecruitingOBSERVATIONAL

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

This study is checking how well a new medicine called garadacimab works for people with a rare condition called Hereditary Angioedema (HAE). HAE causes sudden, painful swelling attacks. We are inviting people who are starting garadacimab treatment in England to join this study. We will follow participants for 4 years, looking at how many swelling attacks they have, if the medicine prevents them, and if there are any side effects. We'll also see how HAE affects their daily life. The aim is to understand how garadacimab works in real-world settings, outside of very controlled trials, to help doctors and patients make informed decisions.

At a glance

Status

Recruiting

Sponsor

CSL Behring

Enrolment target

200

Start

21 Jul 2025

Estimated completion

31 Aug 2030

Hereditary Angioedemas

What is this study about?

This study focuses on a condition called Hereditary Angioedema, often shortened to HAE. HAE is a rare genetic condition that causes sudden, unpredictable swelling attacks in different parts of the body, such as the face, hands, feet, tummy, or throat. These attacks can be very painful, debilitating, and, if they affect the throat, potentially life-threatening. A new medication called garadacimab has been developed to help manage HAE.

This study is not testing garadacimab in a new way or asking you to take it differently. Instead, it's an 'observational' study. This means we want to see how garadacimab works for people with HAE who are already starting to take it as part of their usual care, as prescribed by their doctor. We will be observing how well it helps prevent swelling attacks and if it has any side effects when used in day-to-day life, rather than in a strict research environment.

Your participation will help us understand the true benefits and safety of garadacimab for people with HAE in the UK. The information we gather will complement existing knowledge from more controlled clinical trials, giving a fuller picture of how this medicine works when used by many different patients in their normal routines. This will be valuable for doctors and other patients with HAE in the future.

Key takeaways

This study evaluates garadacimab for Hereditary Angioedema (HAE).
It's an observational study, meaning we watch how the medicine works in daily life.
Participation involves sharing medical records and using an electronic diary.
No extra medication or hospital visits are required.
Results will help understand garadacimab's real-world effectiveness and safety.

Who may be eligible?

To join this study, you need to be at least 12 years old. You must have been diagnosed with Hereditary Angioedema (HAE) by a doctor. Importantly, you should be about to start taking the medicine garadacimab as part of your regular treatment, decided by your doctor, before you officially join the study.

We also need you to be able to give your consent to take part, or if you're under 18, your parent or guardian needs to give permission for you. You should also be comfortable using an electronic device, like a smartphone or computer, to record some information.

You cannot join this study if you have other types of swelling conditions that are not HAE, or if you are already taking part in another clinical study (especially one that involves garadacimab). If you join this study and then decide to take part in another clinical trial later on, you would need to leave this study.

Quick self-check

Are you 12 years old or older?
Have you been diagnosed with Hereditary Angioedema (HAE)?
Are you about to start taking garadacimab as directed by your doctor?
Can you comfortably use a smartphone or computer for recording information?
Are you NOT currently in another clinical study?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you'll be followed for about four years after you start taking garadacimab. There are no extra hospital visits or special tests for this study. We will collect information from your existing medical records and sometimes during your usual doctor's appointments. You'll also be asked to use an electronic diary (like an app on your phone or computer) to record details about any HAE swelling attacks you might have.

We will look at your history from your medical records and your diary entries for the 3 to 12 months before you enrolled in the study to understand your HAE attacks before garadacimab. Throughout the study, we will continue to monitor your HAE attacks, any other HAE treatments you use, and how often you need to use healthcare services for your HAE.

Potential risks and benefits

The potential benefits of taking part include helping doctors and other patients better understand how garadacimab works in real-life situations. While there are no direct medical benefits from participating, your information could be very valuable for advancing knowledge about HAE treatment. Since you are already starting garadacimab as part of your standard care, there are no additional risks from the medication itself by being in the study. Your records will be kept private, and you are free to leave the study at any time without affecting your medical care.

Locations (22)

Medical Research of Arizona
Phoenix, United States· Recruiting
Sirven and Associates
Miami, United States· Recruiting
Asthma and Allergy Wellness Center
Saint Charles, United States· Recruiting
Family Allergy and Asthma
Louisville, United States· Not yet recruiting
John Hopkins University
Baltimore, United States· Active not recruiting
Washington University
St Louis, United States· Active not recruiting
Riverside Medical Group, Belleville
Belleville, United States· Recruiting
Kepler Universitätsklinikum - Med Campus III.
Linz, Austria· Recruiting
Providence Health Care Society
Vancouver, Canada· Recruiting
Universitaetsklinikum Ulm
Ulm, Germany· Recruiting
HZRM Haemophilie-Zentrum Rhein Main
Frankfurt am Main, Germany· Active not recruiting
Medizinische Hochschule Hannover
Hanover, Germany· Active not recruiting

+10 more sites — see the official record for the full list.

Common questions

What is Hereditary Angioedema (HAE)?

HAE is a rare genetic condition that causes sudden, unpredictable attacks of painful swelling, often in the face, hands, feet, tummy, or throat.

Is garadacimab a new medicine?

Yes, garadacimab is a newer medication that helps prevent HAE swelling attacks. This study is watching how it works in everyday use.

Do I have to take extra medicine for this study?

No, you don't take any extra medicine for the study. You'll only take garadacimab if your doctor has already prescribed it for you as part of your usual care.

How long will I be in the study?

If you join, you will be followed for about four years after you start taking garadacimab.

Will my personal details be kept private?

Yes, all your information will be kept strictly confidential and your privacy will be protected.

How to find out more

Trial Registration Coordinator

clinicaltrials@cslbehring.com +1 610-878-4697 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.