Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Open-Label Safety, Pharmacokinetic, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) with Hereditary Angioedema Type I or II

This important study is investigating a new medication called sebetralstat, also known as KVD900. It's designed for children aged 2 to 11 who have a rare genetic condition called Hereditary Angioedema (HAE), specifically Types I or II. HAE causes sudden, severe swelling episodes. The main goal of this particular study is to confirm how safe sebetralstat is for these young patients. Researchers will be carefully monitoring for any unwanted side effects that might occur while children are taking the medicine. They also want to understand how the medicine works in the body and if it helps to reduce or relieve HAE attacks, making it a potential new treatment option for children living with this challenging condition.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Kalvista Pharmaceuticals Limited

Enrolment target

Start

23 Aug 2024

Hereditary Angioedema Type I or II

What is this study about?

This clinical trial is all about a new medicine called sebetralstat (also known as KVD900) for children with a rare condition called Hereditary Angioedema, or HAE. HAE causes unpredictable and sometimes very serious swelling in different parts of the body. There are different types of HAE, and this study focuses on Types I and II, which are the most common.

The main aim of this study is to check how safe sebetralstat is for children between the ages of 2 and 11 years old. Doctors will be looking very closely at any side effects or unwanted reactions that children might have while taking the medicine. This is a "Phase III" study, which means the medicine has already been tested in earlier stages and now researchers need to gather more information on its safety and how well it works before it can be widely used.

While safety is the top priority, the study will also explore how well sebetralstat helps children with their HAE attacks. This includes looking at how quickly symptoms improve once a child takes the medicine, and how long it takes for an attack to completely go away. They'll also see if children need to use other emergency treatments less often if they are taking sebetralstat. The hope is that this new medicine could offer a helpful way to manage HAE for young patients.

Key takeaways

This study is testing a new medicine called sebetralstat for children (2-11 years old) with HAE Types I or II.
The main goal is to understand how safe the medicine is for these young patients.
Researchers will also see how well sebetralstat helps with HAE attacks.
All children in this study will receive the active medicine.
It's a later-stage study aiming to confirm benefits and risks before potential wider use.

Who may be eligible?

This study is open to both boys and girls who have been diagnosed with Hereditary Angioedema (HAE) Type I or Type II. It's specifically for children in a particular age group.

To join, a child must be at least 2 years old but not older than 11 years old at the time they start the study. There are no other specific restrictions on who can take part mentioned beyond having one of these two types of HAE and being within the age range.

It's important that your child's doctor confirms they have HAE Type I or II, as this is a key requirement for participating. The study team will review all medical information to ensure your child meets all the criteria to safely take part.

Quick self-check

Is your child between 2 and 11 years old?
Has your child been diagnosed with Hereditary Angioedema (HAE) Type I?
Has your child been diagnosed with Hereditary Angioedema (HAE) Type II?
Are you able to attend regular clinic visits for your child?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your child takes part in this study, they will receive the study medicine, sebetralstat (KVD900). Since this is a medicine that needs to be taken regularly, there will be a schedule of follow-up appointments. These visits are important for the medical team to monitor your child's health, check for any side effects, and see how the medicine is working. You will likely have regular clinic visits where your child will have physical examinations, and blood samples may be taken to understand how their body handles the medicine.

You and your child will be asked to keep a record of any HAE attacks and how your child feels, which helps the researchers understand the medicine's effectiveness. The total length of your child's participation in the study isn't specified here, but clinical trials usually involve several months to over a year of follow-up to gather enough information. You will receive clear instructions on how and when to give the medicine, and what to do if an HAE attack occurs.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit could be that sebetralstat helps to reduce the frequency or severity of your child's HAE attacks, or provides quicker relief from symptoms. Your child would also receive careful medical attention throughout the study. Potential risks include experiencing side effects from the new medicine; these could range from mild to more serious, and researchers will be monitoring for these very closely. There's also the chance that the medicine might not work for your child. It is very important to remember that your child can stop participating in the study at any time, for any reason, without affecting their future medical care.

Locations (3)

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Italy
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Germany
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France

Common questions

What is Hereditary Angioedema (HAE)?

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

What is sebetralstat (KVD900)?

Sebetralstat is a new medicine being studied to help prevent or treat the swelling attacks in people with HAE. It's an oral medication, meaning it's taken by mouth.

Why are they studying this medicine in children?

It's important to find safe and effective treatments specifically designed for children, as their bodies process medicines differently than adults. This study aims to confirm if sebetralstat is suitable for young patients.

Will my child definitely get the new medicine?

Yes, in this particular study, all participating children will receive sebetralstat (KVD900). This type of study is called 'open-label', meaning everyone knows what medicine is being given.

What does 'Phase III' mean for a clinical trial?

Phase III is a later stage of clinical research. It means the medicine has already shown promise in earlier studies, and now doctors need more information from a larger group of patients to confirm its safety and how well it works before it can be approved for wider use.