The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to \< 12 years of age for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).
Who can participate
Age range
2 Years – 11 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Area Under the Plasma Concentration-Time Curve From Time 0 to the Last Measurable Concentration (AUC0-last) of Berotralstat
Timeframe: Week 2
2
Area Under the Plasma Concentration-Time Curve From Time 0 to 6 Hours Post-dose (AUC0-6) of Berotralstat
Timeframe: Predose and up to 6 hours post dose at Week 2
3
Concentration at the End of the Dosing Interval (Ctrough) of Berotralstat
Timeframe: Predose at Week 2
4
Maximum Observed Plasma Concentration (Cmax) of Berotralstat
Timeframe: Predose and up to 6 hours post dose at Week 2
5
Time of Last Measurable Plasma Concentration (Tlast) of Berotralstat
Timeframe: Predose and up to 6 hours post dose at Week 2
6
Time to Maximum Plasma Concentration (Tmax) of Berotralstat
Timeframe: Predose and up to 6 hours post dose at Week 2