Berotralstat Treatment in Children With Hereditary Angioedema

Phase 3 Active not recruiting 29 enrolled BioCryst Pharmaceuticals

Non-randomizedSingle-groupOpen-labelPrevention

Summary

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).

Timeline

Start: 2022-10-25
Primary completion: 2024-09-11
Completion: 2027-02

Publications

Bernatoniene J, Bourgoin-Heck M, Cancian M, Yang W, Hagin D, Pagnier A, Stobiecki M, Kinaciyan T, Phillips-Angles E, Gayet S, Bara NA, Hunter J, Mateescu E, DeSpirito M, Johnston D, Long D, Iocca H, Petroni D, Aygoren-Pursun E. Oral berotralstat for hereditary angioedema prophylaxis in patients aged 2 to <12 years: APeX-P interim results. Ann Allergy Asthma Immunol. 2025 Dec;135(6):681-688.e3. doi: 10.1016/j.anai.2025.07.012. Epub 2025 Jul 25.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	berotralstat	Small molecule	78 mg	Oral
Subject	berotralstat	Small molecule	96 mg	Oral
Subject	berotralstat	Small molecule	108 mg	Oral
Subject	berotralstat	Small molecule	150 mg	Oral

Berotralstat Treatment in Children With Hereditary Angioedema

Summary

Timeline

Publications

Drugs

Indications