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).
Eligibility
Sex
ALL
Min age
2 Years
Max age
11 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Area Under the Plasma Concentration-Time Curve From Time 0 to the Last Measurable Concentration (AUC0-last) of Berotralstat — Week 2 AUC0-last is the area under the plasma concentration-time curve from time 0 to the time of the last measurable concentration.
Area Under the Plasma Concentration-Time Curve From Time 0 to 6 Hours Post-dose (AUC0-6) of Berotralstat — Predose and up to 6 hours post dose at Week 2 AUC0-6 is the area under the plasma concentration-time curve from time 0 to 6 hours.
Concentration at the End of the Dosing Interval (Ctrough) of Berotralstat — Predose at Week 2 Ctrough is the concentration at the end of a dosing interval of berotralstat.
Maximum Observed Plasma Concentration (Cmax) of Berotralstat — Predose and up to 6 hours post dose at Week 2 Cmax is the maximum observed plasma concentration of berotralstat.
Time of Last Measurable Plasma Concentration (Tlast) of Berotralstat — Predose and up to 6 hours post dose at Week 2 Tlast is the time of the last measurable concentration (Clast) of berotralstat collected over the sampling interval.
Time to Maximum Plasma Concentration (Tmax) of Berotralstat — Predose and up to 6 hours post dose at Week 2 Tmax is the time taken to reach the maximum observed plasma concentration of berotralstat.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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