Not recruitingRare disease

Study on the Safety and Effects of Garadacimab for Preventing Hereditary Angioedema in Children Aged 2 to 11

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Hereditary Angioedema (HAE), a rare genetic condition that causes sudden swelling in various parts of the body. The study is testing a treatment called garadacimab, also known by its code name CSL312. This treatment is given as a solution for injection under the skin, known as subcutaneous use. The purpose of the study is to evaluate the safety and how the body processes this medication in children aged 2 to 11 years who have HAE.

Participants in the study will receive regular injections of garadacimab over a period of up to 12 months. The study will monitor the number of HAE attacks the participants experience and any side effects that may occur. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment. The study aims to understand how well garadacimab works in preventing HAE attacks and to ensure it is safe for use in children.

Throughout the study, researchers will collect information on the frequency and severity of HAE attacks, as well as any adverse events or side effects. This information will help determine the effectiveness of garadacimab in reducing the number of HAE attacks and improving the quality of life for children with this condition. The study is expected to continue until mid-2026, providing valuable insights into the treatment of Hereditary Angioedema in young patients.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the patient will be assessed to ensure they meet the inclusion criteria. This includes being between 2 to 11 years old, having a body weight at or above the 10th percentile for their age, and having a confirmed diagnosis of hereditary angioedema (HAE). The patient must have experienced at least two HAE attacks in the six months prior to the screening.
Step 2
Treatment administration
The patient will receive the medication garadacimab, which is a solution for injection. The administration route is subcutaneous, meaning the injection is given under the skin.
The study aims to evaluate the safety and how the body processes the medication when used as a preventive treatment for HAE in children.
Step 3
Monitoring and evaluation
Throughout the study, the patient will be monitored for any treatment emergent adverse events (TEAEs). This includes tracking the number and percentage of subjects experiencing these events, as well as the rates per injection and per subject year.
The study will also measure the maximum concentration of the medication in the body at steady-state, the trough concentration, and the time it takes to reach maximum concentration.
Step 4
Assessment of efficacy
The study will assess the time-normalized number of HAE attacks per month and per year, including those treated with on-demand treatment. It will also evaluate the number of moderate or severe attacks and the percentage reduction in attacks.
The goal is to determine the number of subjects experiencing significant reductions in attack frequency, including those who become attack-free.
Step 5
Completion of the study
The study is estimated to end on June 11, 2026. During this time, data will be collected to evaluate the safety and effectiveness of the treatment in preventing HAE attacks in pediatric patients.

Who can join the trial?

4 criteria

Can be either a boy or a girl.
Must be between 2 and 11 years old, and have a body weight that is at least in the 10th percentile for their age. This means their weight should be similar to or more than most children their age.
Must have a diagnosis of **Hereditary Angioedema (HAE)**, which is a condition that can cause sudden swelling in different parts of the body.
Must have had at least 2 episodes of HAE attacks in the 6 months before the study starts. An HAE attack is when the swelling happens.

Who cannot join the trial?

10 criteria

Patients who have any other serious health conditions that could interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent infection or illness that could affect the study results.
Patients who are allergic to any of the study medications or their ingredients.
Patients who are unable to follow the study procedures or instructions.
Patients who have a history of drug or alcohol abuse.
Patients who are pregnant or breastfeeding.
Patients who have received certain medications or treatments that could interfere with the study.
Patients who have a history of certain blood disorders.
Patients who have had a recent surgery or medical procedure.

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Verified sites

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ASST Fatebenefratelli Sacco
Milan, Italy
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Azienda Ospedaliera di Padova
Padua, Italy
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Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania
Catania, Italy

Trial locations

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Investigated drugs

CSL312 (Garadacimab) is a medication being studied for its ability to prevent hereditary angioedema (HAE) attacks in children aged 2 to 11. It is given as an injection under the skin and works by targeting specific proteins in the blood that are involved in causing the swelling attacks associated with HAE. The study aims to understand how safe and effective this medication is for young patients and how it behaves in their bodies.

Garadacimab

What is already known about the treatment

Garadacimab – This medication is administered subcutaneously and is currently being studied in clinical trials for its safety and effectiveness in treating hereditary angioedema (HAE) in children aged 2 to 11. It is in the investigational stage, with ongoing research to understand its full potential and safety profile. Garadacimab is primarily indicated for the prophylactic treatment of HAE, a condition characterized by recurrent episodes of severe swelling. At the molecular level, it works by inhibiting a specific protein involved in the inflammatory process, thereby reducing the frequency and severity of swelling attacks. It is classified pharmacologically as a monoclonal antibody.

Investigated diseases

Hereditary Angioedema – Hereditary angioedema is a genetic disorder characterized by recurrent episodes of severe swelling, often affecting the limbs, face, intestinal tract, and airway. The swelling is caused by a deficiency or dysfunction of a protein called C1 inhibitor, which normally helps regulate inflammation and swelling in the body. These episodes can be triggered by stress, trauma, or even spontaneously without a clear cause. The swelling episodes can last for several days and may vary in frequency and severity among individuals. It is important to note that hereditary angioedema is not caused by allergies and does not respond to typical allergy treatments. The condition is inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder.