Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Lifeline for HAE patients: study offers continued access to promising treatment

NCT ID NCT04933721

Disease control Sponsor: BioCryst Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study gives people with hereditary angioedema (HAE) who were in earlier berotralstat trials continued access to the medication. About 139 participants will receive berotralstat and be monitored for side effects. The goal is to keep treating their condition while gathering more safety information.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 139 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2021

Expected to finish

Aug 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Males and females currently enrolled in BioCryst-sponsored Study 302, 204, or 304 * Participant or parent/legally designated representative (for participants \<18 years of age) able to provide written informed consent. * Would benefit from continued berotralstat treatment * Acceptable effective contraception Key Exclusion Criteria: * Pregnancy or breast-feeding * Any condition or situation, including medical history that, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study * Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study. * Use of any other investigational medicinal product at the Baseline visit or any time during the study.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for HAE are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

hereditary angioedema

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HAE Hereditary angioedema

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    16 sites in 11 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Study Center

    Ottawa, Canada

  • Study Center

    Brno, Czechia

  • Study Center

    Pilsen, Czechia

  • Study Center

    Grenoble, France

  • Study Center

    Marseille, France

  • Study Center

    Paris, France

  • Study Center

    Frankfurt, Germany

  • Study Center

    Skopje, North Macedonia

  • Study Center

    Krakow, Poland

  • Study Center

    Martin, Slovakia

  • Study Center

    Cape Town, South Africa

  • Study Center

    Daegu, South Korea

  • Study Center

    Gwangju, South Korea

  • Study Center

    Seoul, South Korea

  • Study Center

    Madrid, Spain

  • Study Center

    Bristol, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.