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Study on the Safety and Effects of Berotralstat for Children Aged 2 to 12 with Hereditary Angioedema (HAE)

Verified siteRegistered drugNo placebo
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What is this trial about?

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

This clinical trial is focused on studying a condition called Hereditary Angioedema (HAE), which is a rare genetic disorder that causes sudden swelling in various parts of the body. The study is testing a medication called Berotralstat, which is taken orally to help prevent these swelling attacks. The medication is available in two forms: hard capsules known as Orladeyo 150 mg and granules, both containing the active substance Berotralstat. The purpose of the study is to evaluate the safety and how the body processes this medication in children aged 2 to less than 12 years who have HAE.

Participants in the study will take Berotralstat over a period of time, and researchers will monitor them to see how the medication affects their bodies and if it helps reduce the frequency and severity of HAE attacks. The study will also look at any side effects that may occur. The medication is taken by mouth, and the study will last for a maximum of 144 weeks. During this time, researchers will collect information on the number of HAE attacks, how long they last, and any other related symptoms.

The study aims to provide valuable information on the use of Berotralstat in young children with HAE, helping to understand its potential benefits and any risks involved. This information could be important for developing better treatment options for children with this condition in the future.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the participant will be assessed to ensure they meet the eligibility criteria. This includes being between 2 to less than 12 years old, weighing at least 12 kg, and having a clinical diagnosis of hereditary angioedema (HAE).

  2. Step 2

    Medication administration

    The participant will receive berotralstat, which is administered orally. The specific form used in this study is a 150 mg hard capsule known as Orladeyo.

    The medication is taken once daily to help prevent HAE attacks.

  3. Step 3

    Monitoring and assessments

    Throughout the study, the participant will undergo regular monitoring to evaluate the safety and effectiveness of the medication. This includes checking for any side effects, measuring height and weight, and conducting physical examinations.

    Blood tests and other laboratory analyses will be performed to assess the participant's health and the medication's impact.

  4. Step 4

    Evaluation of effectiveness

    The study will track the frequency and severity of HAE attacks, the duration of symptoms, and any treatments needed for acute attacks.

    The participant's response to the medication will be evaluated over time, with assessments occurring from Week 1 through Weeks 12 and 48.

  5. Step 5

    Completion of the study

    The study is expected to conclude by February 26, 2027. At the end of the study, a final assessment will be conducted to determine the overall safety and effectiveness of berotralstat in preventing HAE attacks in children.

Who can join the trial?

7 criteria

  • Children who are boys or girls, aged 2 to less than 12 years old, and weigh at least 12 kilograms (about 26 pounds).
  • A parent or caregiver must be willing and able to sign a written consent form, and the child may need to agree as well, if appropriate.
  • The child must have a clinical diagnosis of Hereditary Angioedema (HAE). This means they have specific test results or a family history that confirms they have HAE.
  • If the child is not currently receiving regular treatment to prevent HAE attacks, they must have had at least 2 HAE attacks in the 6 months before joining the study.
  • The child must have access to and be able to use medications approved for treating sudden HAE attacks.
  • The study doctor must believe that the child would benefit from taking a long-term oral medication to prevent HAE attacks.
  • Girls who have started their menstrual periods and boys must either not be sexually active or, if they are or become sexually active during the study, they must agree to use effective birth control methods.

Who cannot join the trial?

3 criteria

  • Children who are not between 2 to less than 12 years old cannot participate.
  • Children who weigh less than 12 kg cannot participate.
  • Children who do not have **Hereditary Angioedema (HAE)** cannot participate. HAE is a rare condition that causes sudden swelling in different parts of the body.
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Investigated drugs

Berotralstat is a medication being studied for its ability to prevent attacks in children with hereditary angioedema (HAE). It is taken by mouth and the study aims to understand how the body processes this medication in young children.

What is already known about the treatment

Berotralstat – This medication is administered orally and is currently being studied in a Phase 3 clinical trial for its safety and pharmacokinetics in children with Hereditary Angioedema (HAE) aged 2 to under 12 years. It is used as a prophylactic treatment to prevent attacks of HAE, a rare genetic condition that causes severe swelling. Berotralstat works by inhibiting a specific enzyme involved in the production of bradykinin, a molecule that contributes to the swelling and pain associated with HAE attacks. It is classified as a kallikrein inhibitor, which helps reduce the frequency and severity of these attacks.

Investigated diseases

Hereditary Angioedema – Hereditary Angioedema (HAE) is a rare genetic disorder characterized by recurrent episodes of severe swelling. This swelling can occur in various parts of the body, including the hands, feet, face, and airway. The condition is caused by a deficiency or dysfunction of a protein called C1 inhibitor, which leads to an overproduction of bradykinin, a peptide that increases blood vessel permeability. Swelling episodes can be painful and may last for several days. The frequency and severity of these episodes can vary widely among individuals. HAE is typically inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2024-511257-22-00Protocol codeBCX7353-304Estimated enrolment29 patientsSponsorBiocryst Pharmaceuticals Inc.

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