Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3 Open-label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema in Pediatric Subjects 2 to 11 Years of Age

This research study is looking at a new medicine called garadacimab for children between 2 and 11 years old who have hereditary angioedema (HAE). HAE is a rare condition that causes sudden swelling attacks. The main goals of the study are to understand how safe this medicine is for children and how well it works to prevent these swelling attacks. Researchers will carefully watch for any side effects, how the medicine acts in the body, and if it reduces the number of HAE attacks. This is a 'Phase 3' study, which means the medicine has already been tested in earlier stages and is now being looked at in more detail in a larger group of people to confirm its benefits and risks.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

CSL Behring LLC

Enrolment target

Start

17 Oct 2023

Hereditary angioedema (HAE)

What is this study about?

This study is about a new medicine called garadacimab for children aged 2 to 11 years who have a rare condition called Hereditary Angioedema, or HAE. HAE causes unpredictable and often painful episodes of swelling in different parts of the body. These swelling attacks can be serious, affecting areas like the face, throat, gut, and limbs.

The main idea behind this study is to see if garadacimab can help prevent these HAE attacks in children. This type of treatment is called 'prophylactic,' which simply means it's given regularly to stop problems before they start. Researchers want to make sure the medicine is safe for young people and that it works effectively to reduce the number and severity of attacks.

To do this, they will carefully monitor several things. They'll track any side effects – these are called 'adverse events' – to understand how well children tolerate the medicine. They will also measure how the medicine moves through the body and how it affects the body over time. Importantly, they will count how many HAE attacks children have while on the medicine, to see if garadacimab can significantly lower this number compared to not taking it.

Key takeaways

This study tests a new medicine (garadacimab) for children with HAE.
It aims to prevent HAE attacks and assess the medicine's safety.
Children aged 2 to 11 with HAE may be eligible.
Participation involves regular clinic visits and monitoring for side effects.
It's a Phase 3 study, meaning the medicine is in advanced testing.

Who may be eligible?

To be considered for this study, you would need to be the parent or guardian of a child with hereditary angioedema (HAE). Children who might take part in this study are between 2 and 11 years of age.

While the study description doesn't list many specific criteria, generally, children would need a confirmed diagnosis of HAE. There might also be other health requirements to make sure the medicine is safe for your child. For example, certain other medical conditions or medicines your child is taking might mean they can't join.

It's very important to discuss all of your child's health history and current medications with the study doctor to find out if they are a good fit for this research. They will have a full list of specific requirements.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your child between 2 and 11 years old?
Does your child have a confirmed diagnosis of hereditary angioedema (HAE)?
Are you able to attend regular clinic visits for your child?
Is your child currently taking any other medicines for HAE or other conditions (you'll need to discuss these fully)?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they would receive the study medicine, garadacimab. This medicine is given as an injection. The study team will explain how and when these injections will be given. Your child would have regular visits to the clinic for check-ups. During these visits, doctors and nurses would perform various tests, like blood tests, to see how the medicine is working and to monitor for any side effects. They would also ask you to keep a diary or record of your child's HAE attacks and any symptoms they experience. The full length of your child's participation in the study, including follow-up appointments after the main treatment period, would be explained to you by the study team.

Potential risks and benefits

Taking part in a study like this means your child might receive a new medicine that could potentially reduce their HAE attacks. However, as with any medicine, there's always a chance of side effects. These could be mild, like a reaction at the injection site, or in rare cases, they could be more serious. The study team will closely monitor your child for any problems and explain all known risks. Remember, you have the right to withdraw your child from the study at any time, for any reason, without it affecting their regular medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany
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Unverified
Italy

Common questions

What is hereditary angioedema (HAE)?

HAE is a rare genetic condition that causes sudden and unpredictable swelling attacks in different parts of the body, like the hands, feet, face, and throat.

What is garadacimab?

Garadacimab is a new medicine being tested in this study to see if it can help prevent HAE attacks.

What does 'Phase 3' mean?

Phase 3 means the medicine has already passed earlier safety tests and is now being studied in a larger group of people to confirm its benefits and check for any less common side effects.

Will my child's HAE attacks be tracked?

Yes, a key part of the study is to record how many HAE attacks your child has and how severe they are, to see if the medicine helps.

What are 'adverse events'?

Adverse events are any unwanted medical problems that happen during the study. The researchers will carefully record all of these, whether they seem related to the medicine or not.