Study on the Safety and Effects of Sebetralstat for Children Aged 2-11 with Hereditary Angioedema Type I or II

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What is this study about?

This clinical trial is focused on studying a condition called Hereditary Angioedema (HAE), specifically types I and II, in children aged 2 to 11 years. HAE is a rare genetic disorder that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The trial will use a medication known as sebetralstat, which is also referred to by its code name KVD900. This medication is taken orally as an orodispersible tablet, meaning it dissolves in the mouth without the need for water.

The purpose of the study is to evaluate the safety and tolerability of sebetralstat in young patients with HAE. Participants in the study will receive the medication and be monitored over a period of up to 12 weeks. During this time, researchers will observe any side effects and how well the medication is tolerated by the children. The study will also look at how quickly symptoms improve after taking the medication and how long it takes for an HAE attack to resolve.

Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of sebetralstat. The trial aims to gather important information that could help in understanding how this medication works in children with HAE and potentially improve treatment options for this condition in the future.

1 joining the study

Upon joining the study, the patient will be assessed to ensure they meet the eligibility criteria. This includes being between 2 to 11 years old, having a confirmed diagnosis of hereditary angioedema (HAE) Type I or II, and having experienced at least one HAE attack in the past year.

The caregiver must be able to store and administer the investigational medication properly and complete a diary as required.

2 medication administration

The patient will receive sebetralstat, also known as KVD900, in the form of an orodispersible tablet. This means the tablet dissolves in the mouth without the need for water.

The medication is taken orally as needed during an HAE attack. The specific dosage and frequency will be determined by the study protocol and the healthcare provider.

3 monitoring and assessment

Throughout the study, the patient will be monitored for any side effects or adverse events. This includes any negative reactions to the medication, regardless of whether other medications are used or if sebetralstat is discontinued.

The study aims to evaluate the time it takes for symptoms to begin to improve and for the HAE attack to resolve after taking the medication.

4 completion of the study

The study is expected to continue until August 2027. During this time, the patient’s response to the medication and any side effects will be documented.

The goal is to assess the safety and effectiveness of sebetralstat in managing HAE attacks in pediatric patients.

Who Can Join the Study?

The patient must be a boy or girl between 2 and 11 years old.
The patient must have a confirmed diagnosis of Hereditary Angioedema (HAE) Type I or II. This means they have a specific condition that affects their blood vessels and can cause swelling.
The patient must have had at least one HAE attack in the past year. An attack is when the symptoms of the condition appear.
The person taking care of the patient, called a caregiver, must be able to store and give the study medicine correctly. They also need to be able to read, understand, and fill out a diary about the patient’s condition.
The doctor in charge of the study must believe that the patient and their caregiver are willing and able to follow all the study rules.
The patient’s parent or legal representative must sign a form agreeing to the study, and the patient must agree to participate if they are old enough to understand.

Who Cannot Join the Study?

Patients who do not have Hereditary Angioedema Type I or II cannot participate. This is a condition that causes sudden swelling in different parts of the body.
Patients who are not between the ages of 2 and less than 12 years old cannot participate.
Patients who are not able to safely take the study medication cannot participate.
Patients who have other medical conditions that might interfere with the study cannot participate.
Patients who are taking other medications that might interfere with the study cannot participate.
Patients who have had a recent illness or infection that might interfere with the study cannot participate.
Patients who have a history of not following medical advice or treatment plans cannot participate.
Patients who are part of another clinical trial cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Centre Hospitalier Universitaire De Lille	Lille	France
Aix Marseille University	Marseille	France
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA	Rome	Italy

Other Sites

Site Name	City	Country
Azienda Ospedaliera di Padova	Padua	Italy
IRCCS Policlinico San Donato	San Donato Milanese	Italy
Assistance Publique Hopitaux De Paris	Paris	France
HZRM Haemophilie-Zentrum Rhein Main GmbH	Mörfelden-Walldorf	Germany
Gsmwrf Unojpiocrm Fqwiwjfkk	Frankfurt	Germany

Trial status

Country	Status	Recruitment Start
France	Not recruiting	01.09.2024
Germany	Not recruiting	01.09.2024
Italy	Not recruiting	01.09.2024

Trial locations

Investigated drugs:

Sebetralstat

Sebetralstat is a medication being studied for its safety and effectiveness in children aged 2 to 11 who have Hereditary Angioedema (HAE) Type I or II. This condition causes sudden swelling in different parts of the body. The trial aims to see how well children tolerate this medication and how it behaves in their bodies.

Hereditary Angioedema Type I or II – Hereditary Angioedema (HAE) is a genetic disorder characterized by recurrent episodes of severe swelling, known as angioedema. This swelling can affect various parts of the body, including the limbs, face, intestinal tract, and airway. Type I is the most common form and is caused by low levels of a protein called C1 inhibitor, while Type II is due to dysfunctional C1 inhibitor. These episodes can be triggered by stress, trauma, or other factors, but often occur without a clear cause. The swelling episodes can be painful and may last for several days. HAE is a lifelong condition that typically begins in childhood or adolescence.

Trial ID:

2023-507591-44-00

Protocol code:

KVD900-303

NCT ID:

NCT06467084

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Safety and Effects of Sebetralstat for Children Aged 2-11 with Hereditary Angioedema Type I or II

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?