A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children with Hereditary Angioedema (HAE) Who Are 2 To < 12 Years of Age.

Conditions

Hereditary Angioedema (HAE)

Brief summary

PK : The primary endpoint is the characterization of the PK profile of berotralstat in subjects aged 2 to < 12 years.

Detailed description

Safety : The frequency and severity of adverse events (AEs) and serious adverse events (SAEs), laboratory analyses (clinical chemistry, hematology, coagulation), height, weight, vital signs, electrocardiograms (ECGs), and physical examination findings, Effectiveness: The frequency of attacks, duration of symptoms, anatomical location of attack, on-demand treatment, number of days with angioedema symptoms, assessment of attack severity, discontinuations due to lack of efficacy, and number of hospitalizations and clinic visits from Week 1 through Weeks 12 and 48

Jolanta Bernatoniene, M. Bourgoin‐Heck, Mauro Cancian, William Yang, David Hagin et al. (19 authors)

Annals of Allergy Asthma & Immunology2025-07-253 citations

10.1016/j.anai.2025.07.012

Interventions

DRUGBerotralstat

DRUGOrladeyo 150 mg hard capsules

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
PK : The primary endpoint is the characterization of the PK profile of berotralstat in subjects aged 2 to < 12 years.	—

Secondary

Measure	Time frame
Safety : The frequency and severity of adverse events (AEs) and serious adverse events (SAEs), laboratory analyses (clinical chemistry, hematology, coagulation), height, weight, vital signs, electrocardiograms (ECGs), and physical examination findings, Effectiveness: The frequency of attacks, duration of symptoms, anatomical location of attack, on-demand treatment, number of days with angioedema symptoms, assessment of attack severity, discontinuations due to lack of efficacy, and number of hospitalizations and clinic visits from Week 1 through Weeks 12 and 48	—

Measure

Time frame

Safety : The frequency and severity of adverse events (AEs) and serious adverse events (SAEs), laboratory analyses (clinical chemistry, hematology, coagulation), height, weight, vital signs, electrocardiograms (ECGs), and physical examination findings, Effectiveness: The frequency of attacks, duration of symptoms, anatomical location of attack, on-demand treatment, number of days with angioedema symptoms, assessment of attack severity, discontinuations due to lack of efficacy, and number of hospitalizations and clinic visits from Week 1 through Weeks 12 and 48

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Countries

Austria, France, Germany, Italy, Poland, Spain

Outcome results

None listed