Active not recruitingPHASE3INTERVENTIONAL

Berotralstat Treatment in Children With Hereditary Angioedema

This study is investigating a new medicine called berotralstat for children between 2 and 11 years old who have a rare condition called hereditary angioedema (HAE). HAE causes sudden, severe swelling episodes. The main goals are to figure out the best dose of berotralstat for children based on their body weight, to make sure it's safe for them, and to see how effective it is at stopping these swelling attacks before they start. This research is important because it could lead to a new way to prevent HAE attacks in young children, helping them live more normal lives.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

BioCryst Pharmaceuticals

Enrolment target

Start

25 Oct 2022

Estimated completion

01 Feb 2027

Hereditary Angioedema Pediatric

What is this study about?

This clinical trial is all about a medicine called berotralstat, and how it might help children aged 2 to 11 who have hereditary angioedema (HAE). HAE is a rare genetic condition that causes unpredictable and often severe swelling attacks anywhere in the body, which can be very distressing for children and their families.

The main purpose of this study is to find out the best way to give berotralstat to children. This means figuring out the right dose based on their weight, ensuring it's safe for them, and checking how well it works to prevent those swelling attacks. Researchers are particularly interested in understanding how the medicine is absorbed and used by a child's body.

Ultimately, the hope is that berotralstat could become a regular treatment option to help children with HAE avoid attacks, improving their quality of life and reducing the worry associated with this condition. This kind of preventative treatment is often called 'prophylactic treatment'.

Key takeaways

Tests berotralstat to prevent HAE attacks in children aged 2-11.
Aims to find the safest and most effective dose based on weight.
Involves treatment with berotralstat for over two years.
Requires careful monitoring with regular clinic visits and tests.
Aims to improve preventative treatment options for children with HAE.

Who may be eligible?

To be considered for this study, children need to be between 2 and 11 years old and weigh at least 12 kilograms (about 26 pounds). They must have a clear diagnosis of hereditary angioedema (HAE), and their doctor should believe that long-term preventative treatment for HAE would be beneficial for them. If a child isn't already on preventative HAE treatment, they should typically have had at least two HAE attacks in the six months before joining the study.

There are also some reasons why a child might not be able to join. For example, if they have another type of angioedema, or a family history of sudden heart issues at a young age, they wouldn't be eligible. Certain issues with kidney or liver function, or abnormal heart test results (like an ECG), would also prevent participation.

Additionally, if a child is already taking part in another study involving a new medicine or has done so within the last month, they wouldn't be able to join this particular trial. These criteria are in place to ensure the safety of all participants and to make sure the study results are clear and reliable.

Quick self-check

Is your child aged between 2 and 11 years old?
Does your child weigh at least 12 kg (about 26 pounds)?
Does your child have a confirmed diagnosis of hereditary angioedema (HAE)?
Has your child had at least two HAE attacks in the last six months (if not on current preventative treatment)?
Does your child have generally good health otherwise, with no significant kidney, liver, or heart problems?
Is your child not currently in another study for a new medicine, or has not been within the last month?

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

What does participation involve?

If your child is eligible and you agree for them to take part, the study will involve several stages. Initially, there's a period of at least 12 weeks where your child's usual care for HAE will continue to be monitored. Following this, your child will begin taking the berotralstat medication.

This berotralstat treatment period is split into three parts: the first part lasts 12 weeks, followed by a second part of 36 weeks, and then a third part that lasts 96 weeks. During these periods, your child will receive berotralstat according to their weight. There will be regular visits to the clinic for safety checks, blood tests to see how the medicine is working in their body, and to monitor how well it's preventing HAE attacks. Your child will be in one of four groups based on their weight, with the dosing being carefully adjusted.

The entire study could last approximately two and a half years, including the initial monitoring period and all three phases of berotralstat treatment. Throughout this time, the research team will closely monitor your child's health and the effectiveness of the treatment.

Potential risks and benefits

Participating in a clinical trial offers potential benefits, such as access to a new treatment like berotralstat that might help prevent HAE attacks in your child, under close medical supervision. However, there are also potential risks; for example, the medicine might cause side effects, or it might not work as expected for every child. The researchers will conduct thorough safety checks, but not all potential risks are known in advance. It's important to remember that participation is always voluntary, and you have the right to withdraw your child from the study at any time, for any reason, without it affecting their future medical care.

Locations (15)

Investigative Site #1
Vienna, Austria
Investigative Site #1
Ottawa, Canada
Investigative Site #3
Grenoble, France
Investigative Site #2
Marseille, France
Investigative Site #1
Paris, France
Investigative Site #1
Berlin, Germany
Investigative Site #2
Frankfurt, Germany
Investigative Site #2
Haifa, Israel
Investigative Site #1
Tel Aviv, Israel
Investigative Site #1
Padova, Italy
Investigative Site #1
Krakow, Poland
Investigative Site #1
Sângeorgiu de Mureş, Romania

+3 more sites — see the official record for the full list.

Common questions

What is hereditary angioedema (HAE)?

HAE is a rare genetic condition that causes sudden and unpredictable attacks of severe swelling in different parts of the body.

What is berotralstat?

Berotralstat is a new medicine being studied to see if it can help prevent HAE attacks in children.

How long will my child be in the study?

Your child is expected to be in the study for a minimum of 12 weeks receiving their usual care, followed by up to 144 weeks (about 2.5 years) taking berotralstat.

Will my child definitely get berotralstat in this study?

Yes, once past the initial monitoring phase, children in this study will receive berotralstat. This is an 'open-label' study, meaning everyone involved knows what treatment is being given.

What kind of tests will my child have?

Your child will have regular check-ups, blood tests to measure the medicine and monitor health, and their HAE attacks will be carefully tracked.