Trial reportAllergy2021
Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial.
Trial report in Allergy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03873116 (A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX7353 as an Oral Treatment for the Prevention of Attacks in Subjects With Hereditary Angioedema), which is not on this map. Cited by 26 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX7353 as an Oral Treatment for the Prevention of Attacks in Subjects With Hereditary Angioedema
Who cites it
26 citing papers in PubMed, 2 syntheses or guidelines pooled it, 45 citations in OpenAlex.
- Network Meta-Analysis of Pharmacological Therapies for Long-Term Prophylactic Treatment of Patients with Hereditary Angioedema.Drugs in R&D · 2025Pooled it
- Interventions for the long-term prevention of hereditary angioedema attacks.The Cochrane database of systematic reviews · 2022Pooled it
- Berotralstat and Health-Related Quality of Life in Hereditary Angioedema: Pooled Analysis of the APeX-2 and APeX-J Trials.Clinical and experimental allergy : journal of the British Society for Allergy and Clinical Immunology · 2026Trial
- Sebetralstat for breakthrough attacks in patients with hereditary angioedema receiving long-term prophylaxis in KONFIDENT-S.The journal of allergy and clinical immunology. Global · 2026Article
- Acquired Angioedema-A Challenge in Medical Practice: A Narrative Review.Journal of clinical medicine · 2026Review
- The International/Canadian hereditary angioedema guideline.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2026Review
- Attack rate reductions following berotralstat initiation among US patients with hereditary angioedema in the real-world.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2026Article
- Berotralstat effectiveness and safety in patients with hereditary angioedema with normal C1 inhibitor.The journal of allergy and clinical immunology. Global · 2026Article
- Reductions in medical visits and hospitalizations following berotralstat initiation in patients with hereditary angioedema in the United States.Journal of managed care & specialty pharmacy · 2025Article
- The significance of chirality in contemporary drug discovery-a mini review.RSC advances · 2024Review
- Advent of oral medications for the treatment of hereditary angioedema.Clinical and translational allergy · 2024Review
- Androgen transition and management of hereditary angioedema long-term prophylaxis in real life: a single-center case series.Orphanet journal of rare diseases · 2024Article
- Letter to the editor: network meta-analysis for indirect comparison of lanadelumab and berotralstat for the treatment of hereditary angioedema.Journal of comparative effectiveness research · 2024Article
- Letter in reply: network meta-analysis for indirect comparison of lanadelumab and for the treatment of hereditary angioedema.Journal of comparative effectiveness research · 2024Article
- Berotralstat for long-term prophylaxis of hereditary angioedema in Japan: Parts 2 and 3 of the randomized APeX-J Phase III trial.The World Allergy Organization journal · 2024Article
- Long-term prophylaxis for hereditary angioedema: Initial experiences with garadacimab and lanadelumab.The journal of allergy and clinical immunology. Global · 2023Article
- Hereditary angioedema with an acute attack resolved after bone marrow transplantation for acute myeloid leukemia: a case report.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2023Article
- FDA-Approved Fluorinated Heterocyclic Drugs from 2016 to 2022.International journal of molecular sciences · 2023Review
- Asymmetric Synthesis of US-FDA Approved Drugs over Five Years (2016-2020): A Recapitulation of Chirality.Pharmaceuticals (Basel, Switzerland) · 2023Review
- Prevention of Recurrent Attacks of Hereditary Angioedema (HAE): Berotralstat and Its Oral Bioavailability.Therapeutics and clinical risk management · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
22 authors at 11 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundWith no approved treatments in Japan for the prevention of hereditary angioedema (HAE) attacks, there is a significant unmet need for long-term prophylactic therapies for Japanese patients with HAE. Berotralstat (BCX7353) is an oral, once-daily, highly selective inhibitor of plasma kallikrein in development for prophylaxis of angioedema attacks in HAE patients.
methodsAPeX-J is a phase 3, randomized, double-blind, placebo-controlled, parallel-group, 3-part trial conducted in Japan (University Hospital Medical Information Network identifier, UMIN000034869; ClinicalTrials.gov identifier, NCT03873116). Patients with a clinical diagnosis of type 1 or 2 HAE underwent a prospective run-in period of 56 days to determine eligibility, allowing enrollment of those with ≥2 expert-confirmed angioedema attacks. Patients were randomly assigned (1:1:1) and stratified by baseline attack rate (≥2 vs. <2 expert-confirmed attacks/month between screening and randomization) to receive once-daily berotralstat 110 mg, berotralstat 150 mg, or placebo. The primary endpoint was the rate of expert-confirmed angioedema attacks during dosing in the 24-week treatment period.
resultsNineteen patients were randomized to receive once-daily berotralstat 110 mg (n = 6), berotralstat 150 mg (n = 7), or placebo (n = 6). Treatment with berotralstat 150 mg significantly reduced HAE attacks relative to placebo (1.11 vs. 2.18 attacks/month, p = .003). The most frequently reported treatment-emergent adverse events (TEAEs) in berotralstat-treated patients (n = 13) were nasopharyngitis (n = 4, 31%), abdominal pain, cough, diarrhea, and pyrexia (n = 2 each, 15%).
conclusionsOrally administered, once-daily berotralstat 150 mg significantly reduced the frequency of HAE attacks and was safe and well tolerated, supporting its use as a prophylactic therapy in patients with type 1 or 2 HAE in Japan.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.