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Long-Term Safety Study of Berotralstat for Patients with Hereditary Angioedema from Previous Trials

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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 known as Hereditary Angioedema (HAE). HAE is a rare genetic disorder that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The treatment being studied in this trial is a medication called Berotralstat, which is taken orally in the form of a capsule. Berotralstat is designed to help prevent the swelling attacks associated with HAE.

The purpose of this study is to monitor the long-term safety of Berotralstat in individuals who have previously participated in Berotralstat studies. Participants in this trial will continue to take Berotralstat over an extended period, allowing researchers to gather important information about its safety and any potential side effects. The study will provide ongoing access to Berotralstat for those who have been part of earlier clinical trials, ensuring they can continue their treatment while contributing to valuable research.

Throughout the study, participants will be monitored for any treatment-related side effects or serious adverse events. The trial will continue until another way to access the medication becomes available, or until the sponsor decides to stop the global development and commercialization of Berotralstat. This study aims to ensure that Berotralstat remains a safe and effective option for managing Hereditary Angioedema in the long term.

The research process

The trial runs in 4 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 must have been previously enrolled in a berotralstat study and not discontinued due to adverse events or noncompliance.

    The participant must provide written informed consent. If aged 12 to 17, an assent form must also be signed.

  2. Step 2

    Medication administration

    The participant will receive berotralstat, which is taken orally.

    The medication is intended for long-term use to monitor safety in individuals with hereditary angioedema.

  3. Step 3

    Monitoring and evaluation

    The study aims to monitor the long-term safety of berotralstat by evaluating the number and proportion of participants experiencing treatment-related adverse events.

    Participants will be observed for any serious adverse events or significant treatment-emergent chemistry abnormalities.

  4. Step 4

    Completion of the study

    The study is estimated to conclude by July 10, 2026, unless another mechanism for drug provision becomes available or the sponsor discontinues the global commercialization and development of berotralstat.

Who can join the trial?

7 criteria

  • Must be a male or a female who is not pregnant or breastfeeding.
  • Must be currently enrolled or previously enrolled in a specific study (BioCryst-sponsored Study 302 or 204) and did not leave the study because of a bad reaction or not following the rules.
  • Must be able to give written permission to join the study. If aged 12 to 17, must be able to read, understand, and agree to sign a form showing they agree to participate, along with a parent or guardian giving permission.
  • Must be considered by the study doctor to benefit from continuing treatment with a medication called berotralstat.
  • Female participants must meet one of these conditions:
    • Be a woman who can have children and agrees to use an effective birth control method during the study.
    • Be a woman who cannot have children, either because she has gone through menopause (no periods for at least 12 months without another medical reason) or has had surgery to remove certain reproductive organs.
    • Must be expected by the study doctor to follow all the study procedures properly.

Who cannot join the trial?

5 criteria

  • Patients who do not have **Hereditary Angioedema**. This is a rare genetic condition that causes sudden swelling in different parts of the body.
  • Patients who are not within the specified age range for the study. The study is open to certain age groups only.
  • Patients who are not part of the specified clinical trial groups. The study is designed for specific groups of people.
  • Patients who are not male or female, as the study includes both genders.
  • Patients who are not considered part of a vulnerable population. This means the study is looking at groups that might need special protection or care.
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Investigated drugs

Berotralstat is a medication used in this clinical trial to help people with hereditary angioedema, a condition that causes sudden swelling in different parts of the body. The trial aims to provide access to this medication for participants who were part of previous studies. The main goal is to ensure that berotralstat is safe for long-term use in managing their condition.

What is already known about the treatment

Berotralstat – Berotralstat is administered orally in the form of a capsule. It is currently being studied in clinical trials to assess its long-term safety for individuals with hereditary angioedema, a rare genetic condition. The medication is primarily indicated for the prevention of attacks in patients with this condition. At the molecular level, berotralstat works by inhibiting plasma kallikrein, an enzyme involved in the production of bradykinin, which is responsible for the swelling and pain associated with hereditary angioedema. It is classified as a kallikrein inhibitor in pharmacology.

Investigated diseases

Hereditary Angioedema – Hereditary Angioedema is a rare genetic disorder characterized by recurrent episodes of severe swelling. This swelling can affect 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 triggered by stress, trauma, or hormonal changes, but they often occur without a clear cause. The swelling can be painful and may last for several days, significantly impacting daily activities. It is important to manage the condition to prevent complications, especially when the swelling affects the airway.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-511285-37-00Protocol codeBCX7353-312Estimated enrolment124 patientsSponsorBiocryst Pharmaceuticals Inc.

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