Not recruitingRare disease

Safety Study of Switching from Current Treatment to Garadacimab in Patients Age 12 and Older with Hereditary Angioedema

Verified siteInvestigationalNo placebo

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What is this trial about?

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

This study focuses on Hereditary Angioedema (HAE), a rare genetic condition that causes sudden episodes of swelling in various parts of the body. The study will evaluate a medication called garadacimab (also known as CSL312), which is given as an injection under the skin. The research aims to assess the safety of switching to garadacimab from other currently used preventive treatments for HAE.

The medication will be administered as a subcutaneous injection with doses up to 400 milligrams per day. The study will involve people who are currently using other preventive treatments for HAE and will monitor how their body responds when switching to garadacimab. The treatment period will last for approximately 3 months.

During the study, participants will receive regular doses of garadacimab, and their health will be closely monitored for any side effects or reactions to the medication. The study will also measure how much of the medication remains in the blood and whether the body produces antibodies against it. Additionally, participants will have the opportunity to share their experience with the new medication compared to their previous treatment.

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
Initial qualification
If you are 12 years or older and have been diagnosed with Hereditary Angioedema (HAE), you may be eligible for this study.
You must have been using lanadelumab, berotralstat, or pdC1INH for HAE prevention at a stable dose for at least 3 months.
Your medical records must confirm your HAE diagnosis through specific laboratory tests.
Step 2
Treatment transition
You will switch from your current HAE prevention medication to garadacimab.
Garadacimab will be administered as an injection under the skin (subcutaneous injection).
Step 3
Monitoring period
Your response to garadacimab will be monitored throughout the study.
Medical staff will track any side effects or reactions that may occur.
Blood samples will be collected to measure garadacimab levels in your body.
The study will check for antibodies your body might develop against garadacimab.
Step 4
Study duration
The study is scheduled to run from August 2025 to June 2026.
During this time, you will be monitored for any changes in your condition and any side effects.
Step 5
Study completion
At the end of the study, you will be asked about your preference regarding garadacimab treatment.
The medical team will evaluate all collected data about your response to the treatment and any side effects.

Who can join the trial?

10 criteria

Must be at least 12 years old when giving consent to participate in the study
Must have previously responded well to treatments for sudden HAE attacks (episodes of swelling)
Must have documented medical records showing one of these conditions:
- A history of swelling episodes without itching or hives
- Low levels of C1INH (a blood protein) - less than 50% of normal
- Low levels of C4 protein in blood tests
- For specific genetic types of HAE: must have confirmed genetic mutations in either FXII or PLG genes, with normal C1INH levels
- Must be currently using one of these preventive treatments for at least 3 months before screening:
  - Lanadelumab
  - Berotralstat
  - pdC1INH (plasma-derived C1 inhibitor)
  - Must be taking a stable and consistent dose of their current preventive treatment

Who cannot join the trial?

10 criteria

Persons younger than 12 years old cannot participate in the study
Individuals who are not currently using approved treatments for hereditary angioedema (HAE)
Patients who are not currently using either KK inhibitor (a type of medication that helps prevent swelling) or pdC1INH (a protein replacement therapy) for HAE prevention
Pregnant women or those planning to become pregnant during the study period
People with severe allergic reactions to similar medications in the past
Individuals participating in other clinical trials
Patients with uncontrolled medical conditions that could interfere with the study
People unable to follow study procedures or attend scheduled visits
Individuals with known substance abuse problems
Patients who have received experimental treatments within the past 30 days

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz
Verified
Mainz, Germany
HZRM Haemophilie-Zentrum Rhein Main GmbH
Verified
Mörfelden-Walldorf, Germany

Trial locations

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Investigated drugs

Garadacimab
(also known as CSL312) is a medication being studied for the treatment of Hereditary Angioedema (HAE). It is designed to prevent HAE attacks in patients who are currently using other preventive treatments. This medication works by targeting specific proteins in the body that are involved in causing swelling episodes in HAE patients.
Plasma-derived C1 inhibitor (pdC1INH)
is a medication made from human blood plasma that helps prevent swelling attacks in people with HAE. It works by replacing the missing or malfunctioning C1 inhibitor protein in patients with HAE.
Kallikrein inhibitors (KK inhibitors)
are medications that work by blocking a specific protein called kallikrein, which is involved in causing swelling in HAE patients. These medications help prevent HAE attacks by reducing the activity of this protein in the body.

Garadacimab

What is already known about the treatment

Garadacimab (CSL312) - A novel investigational medication administered via subcutaneous injection for the prophylactic treatment of Hereditary Angioedema (HAE). This monoclonal antibody works by targeting and inhibiting Factor XIIa, a key protein involved in the cascade that leads to angioedema attacks. Currently undergoing Phase 4 clinical trials, Garadacimab represents a potential alternative to existing HAE prophylactic treatments such as plasma-derived C1 inhibitors and kallikrein inhibitors, aiming to provide long-term prevention of HAE attacks through a different mechanism of action. The medication is being developed as a more convenient treatment option for patients aged 12 years and older who require regular prophylaxis for HAE management.

Investigated diseases

Hereditary Angioedema (HAE) - A rare genetic condition that causes sudden episodes of swelling in various parts of the body, including the hands, feet, face, intestinal walls, and airways. The swelling occurs because of a problem with a blood protein called C1 inhibitor, which normally helps regulate blood vessel function. Episodes of swelling can happen spontaneously or be triggered by stress, injury, or certain medications. The swelling typically develops slowly over several hours and can last for a few days. During an attack, the affected area may feel stretched, painful, and warm to the touch. Between episodes, people with HAE typically have no visible signs of the condition.