Long-term safety study of garadacimab (CSL312) for prevention of hereditary angioedema attacks

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What is this study about?

This clinical trial focuses on studying Hereditary Angioedema, a rare genetic condition that causes severe swelling attacks in different parts of the body. The study examines a new medication called garadacimab (also known as CSL312) that is given as an injection under the skin to prevent these swelling attacks. The purpose is to evaluate how safe and effective this medication is when used for a long time in people who experience regular swelling attacks.

The medication is given as a solution for injection that is administered under the skin (subcutaneous injection). This is a long-term study where participants receive regular treatments with garadacimab to prevent swelling attacks. The study looks at how well the medication works in reducing the number and severity of swelling attacks compared to before treatment.

During the study, researchers track how many swelling attacks occur and how severe they are. They also monitor any side effects that participants may experience while using the medication. The study focuses particularly on seeing if the treatment can significantly reduce the frequency of swelling attacks and improve the quality of life for people with this condition.

1 Initial treatment phase

You will receive garadacimab as a solution for injection under the skin (subcutaneous injection)

The treatment aims to prevent hereditary angioedema attacks (sudden swelling episodes that can occur in different parts of your body)

2 Treatment monitoring

Medical staff will monitor your response to the treatment by tracking:

The number and severity of swelling attacks you experience per month

How often you need additional treatment for sudden attacks

Your personal assessment of how well the treatment is working

3 Safety monitoring

Throughout the study, medical staff will monitor:

Any side effects that may occur after injections

Any serious health changes during treatment

The presence of antibodies your body might develop against the medication

4 Long-term evaluation

The study continues until November 2025

Medical staff will assess:

The reduction in number of swelling attacks compared to before treatment

The percentage of patients who achieve significant reduction in attacks

The overall safety of long-term treatment

Who Can Join the Study?

  • Must be at least 12 years old (male or female)
  • Must have a confirmed diagnosis of hereditary angioedema (HAE) – a rare genetic condition that causes episodes of severe swelling
  • Must have experienced at least 3 HAE attacks in the 3 months before initial screening
  • For patients who have never used the study medication (CSL312): Must complete a run-in period (initial observation period) of at least 1 month
  • During the run-in period, must have experienced an average of at least 1 HAE attack per month

Who Cannot Join the Study?

  • Age below 12 years old
  • Known allergic reactions to CSL312 (the study medication) or similar medications
  • Currently pregnant or breastfeeding women
  • Participation in another clinical trial within the past 30 days
  • History of severe allergic reactions or anaphylaxis (a serious, life-threatening allergic reaction)
  • Significant heart, liver, or kidney disease
  • Active cancer or ongoing cancer treatment
  • Uncontrolled high blood pressure
  • History of blood clotting disorders
  • Current use of medications that could interact with the study drug
  • Mental conditions that could affect the ability to follow study procedures
  • Drug or alcohol abuse within the past year
  • Any condition that, in the opinion of the study doctor, would make participation unsafe
  • Unable or unwilling to comply with study procedures and visits
  • History of serious infections in the past 3 months

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz Mainz Germany
Universitaet Leipzig Leipzig Germany

Other Sites

Site Name City Country Status
Hospital General Universitario Gregorio Maranon Madrid Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Semmelweis University Budapest Hungary
Fakultni Nemocnice U Sv Anny V Brne Brno-Stred Czechia
HZRM Haemophilie-Zentrum Rhein Main GmbH Mörfelden-Walldorf Germany
Fxdqkwfa nfwvygibp Mohbm a Hlzkkpl Prague Czechia
Akexquevv Uva Amsterdam The Netherlands
Gcoywn Ujvptjgoyk Fffcpaqzm Frankfurt Germany
Hucvkhzf Vjki dkfbpfok Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Czechia Czechia
Not recruiting
29.03.2021
Germany Germany
Not recruiting
29.03.2021
Hungary Hungary
Not recruiting
29.03.2021
Spain Spain
Not recruiting
29.03.2021
The Netherlands The Netherlands
Not recruiting
29.03.2021

Trial locations

Investigated drugs:

Garadacimab (also known as CSL312) is an experimental medication designed for preventing attacks in people with hereditary angioedema (HAE). It works by targeting a specific protein in the body that is involved in swelling episodes. The medication is given as an injection under the skin (subcutaneous injection) and is being studied as a preventive treatment to reduce the frequency of HAE attacks.

C1-INH (C1 esterase inhibitor) is a protein that helps regulate various body processes including swelling. In people with hereditary angioedema, there is either not enough C1-INH or it doesn’t work properly. This trial is studying how garadacimab works in patients with this specific type of HAE related to C1-INH deficiency.

Investigated diseases:

Hereditary Angioedema – A rare genetic condition characterized by recurrent episodes of severe swelling in various parts of the body. The swelling typically affects the hands, feet, face, digestive tract, and airways. Episodes can occur spontaneously or be triggered by stress, minor trauma, or other factors. The condition is caused by a deficiency or dysfunction of a blood protein called C1 inhibitor, which helps regulate various body processes. The swelling develops slowly over several hours and usually subsides within 2-5 days. Between episodes, patients may have no visible symptoms.

Trial ID:
2024-510777-18-00
Protocol code:
CSL312_3002
Trial Phase:
Therapeutic confirmatory (Phase III)

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