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New hope for kids with rare swelling disorder: daily pill may stop attacks

NCT ID NCT05453968

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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tests a daily medication called berotralstat in 29 children aged 2 to under 12 who have hereditary angioedema (HAE), a condition that causes sudden, painful swelling attacks. The goal is to find the right dose based on weight and to check if it is safe and effective at preventing these attacks. Participants take the pill daily and are monitored for side effects and attack frequency.

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

29 people

The number who actually took part.

Started

Oct 2022

Expected to finish

Feb 2027

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 to 11 years

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.

Inclusion Criteria: * Male and non-pregnant, non-lactating females 2 to \< 12 years of age * Body weight ≥ 12 kg * Clinical diagnosis of HAE * In the opinion of the investigator, the participant would benefit from long term oral HAE prophylaxis * For subjects who are not currently receiving prophylaxis for HAE, documented history of \>= 2 HAE attacks in the 6 months prior to the enrollment visit. Exclusion Criteria: * Concurrent diagnosis of any other type of recurrent angioedema * Known family history of sudden cardiac death at a young age (\< 40 years of age) * Creatinine clearance using the modified Schwartz formula of ≤ 30 mL/min/1.73 m\^2 * Aspartate aminotransferase or alanine aminotransferase value ≥ 3 × the upper limit of the age-appropriate normal reference range value * Clinically significant abnormal electrocardiogram (ECG) including but not limited to, a corrected QT interval using Fridericia's correction \> 450 msec, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping * Current participation in any other investigational drug study or received another investigational drug within 30 days of enrollment

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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.

Hereditary angioedema Pediatric

Contacts and locations

Locations

  • Investigative Site #1

    Vienna, Ontario, Austria

  • Investigative Site #1

    Ottawa, Ontario, Canada

  • Investigative Site #1

    Paris, France

  • Investigative Site #1

    Berlin, Germany

  • Investigative Site #1

    Tel Aviv, Israel

  • Investigative Site #1

    Padova, Italy

  • Investigative Site #1

    Krakow, Poland

  • Investigative Site #1

    Sângeorgiu de Mureş, Romania

  • Investigative Site #1

    Madrid, Spain

  • Investigative Site #1

    Bristol, United Kingdom

  • Investigative Site #2

    Marseille, France

  • Investigative Site #2

    Frankfurt, Germany

  • Investigative Site #2

    Haifa, Israel

  • Investigative Site #2

    Málaga, Spain

  • Investigative Site #3

    Grenoble, France

More trials for these conditions

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