Evidence map›Paper›PMID 41252457›Full record

ArticleClinical and translational allergy2025

Sebetralstat for On-Demand Treatment of Mucosal Hereditary Angioedema Attacks in KONFIDENT-S.

Jonathan A Bernstein, Emel Aygören-Pürsün, Mauro Cancian, Danny M Cohn, Timothy Craig, Vesna Grivcheva-Panovska, Anthony Jordan, William R Lumry, Inmaculada Martinez-Saguer, Isaac Melamed and 12 more

Registry-linked trialAbstract read
In one paragraph

Article in Clinical and translational allergy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05505916 (An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema Type I or II), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

NCT05505916 phase3completednot on this map

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema Type I or II

TypeinterventionalSponsorKalVista Pharmaceuticals, Ltd.Ran2022 to 2026Enrolled145ConditionsHereditary AngioedemaArmsKVD900 600 mg, KVD900 300 mg
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

22 authors.

Jonathan A BernsteinUniversity of Cincinnati College of Medicine and Bernstein Clinical Research Center, Cincinnati, Ohio, USA.ORCID https://orcid.org/0000-0002-3476-1196
Emel Aygören-PürsünUniversity Hospital Frankfurt, Goethe University Frankfurt, Frankfurt, Germany.
Mauro CancianDepartmental Allergy Division, Department of Systems Medicine, University of Padua, Padua, Italy.
Danny M CohnAmsterdam University Medical Center, University of Amsterdam, Amsterdam, the Netherlands.ORCID https://orcid.org/0000-0002-9036-3723
Timothy CraigDepartment of Medicine, Pediatrics, MFM, and Biomedical Sciences, Penn State University, Hershey, Pennsylvania, USA.ORCID https://orcid.org/0000-0002-7774-4855
Vesna Grivcheva-PanovskaUniversity Clinic of Dermatology, School of Medicine, University Saints Cyril and Methodus, Skopje, North Macedonia.
Anthony JordanDepartment of Clinical Immunology and Allergy, Auckland City Hospital, Te Toka Tumai, Auckland, New Zealand.
William R LumryAARA Research Center, Dallas, Texas, USA.
Inmaculada Martinez-SaguerHZRM Haemophilia Center Rhein Main, Frankfurt, Germany.
Isaac MelamedIMMUNOe Research Center, Centennial, Colorado, USA.
Kazumasa OhmuraInstitute of Preventive Medical Sciences, Health Sciences University of Hokkaido, Hokkaido, Japan.
Jonny PeterDivision of Allergology and Clinical Immunology, Department of Medicine, Groote Schuur Hospital, University of Cape Town, Cape Town, South Africa.ORCID https://orcid.org/0000-0002-2658-0723
Marc A RiedlUniversity of California, San Diego, La Jolla, California, USA.
Daniel F SoteresAsthma & Allergy Associates PC, Colorado Springs, Colorado, USA.
Petra StaubachDepartment of Dermatology and Allergy, University Medical Center, Mainz, Germany.
Marcin StobieckiDepartment of Clinical and Environmental Allergology, Jagiellonian University, Medical College, Krakow, Poland.ORCID https://orcid.org/0000-0003-0161-4920
Ya-Hsiu ChuangKalVista Pharmaceuticals, Framingham, Massachusetts, USA.
Michael D SmithKalVista Pharmaceuticals, Framingham, Massachusetts, USA.
Christopher M YeaKalVista Pharmaceuticals, Framingham, Massachusetts, USA.
Paul K AudhyaKalVista Pharmaceuticals, Framingham, Massachusetts, USA.
Andrea ZanichelliOperative Unit of Medicine, Angioedema Center, IRCCS Policlinico San Donato, Milan, Italy.
Henriette FarkasHungarian Angioedema Center of Reference and Excellence, Department of Internal Medicine and Haematology, Semmelweis University, Budapest, Hungary.

Funding

KalVista Pharmaceuticals
6 · The paper itself

Abstract

backgroundHereditary angioedema (HAE-C1INH) attacks involving mucosal tissue may progress rapidly and often lead to substantial morbidity. Severe laryngeal attacks can be fatal without prompt administration of on-demand treatment. This prespecified interim analysis evaluated the safety and effectiveness of sebetralstat in laryngeal and abdominal attacks in the ongoing, 2-year, open-label extension KONFIDENT-S study (NCT05505916).

methodsEligible participants ≥ 12 years with HAE-C1INH self-administered sebetralstat 600 mg film-coated tablets with an optional second dose after 3 h, if warranted. PRIMARY OUTCOME: incidence of treatment-emergent adverse events (TEAEs). SECONDARY OUTCOMES: times to beginning of symptom relief, reduction in severity, and complete resolution.

resultsAt data cutoff (Sep 14, 2024), 32 laryngeal (16 participants) and 533 abdominal only attacks (102 participants) were treated with sebetralstat. Seven (43.8%) participants with laryngeal attacks experienced 14 TEAEs whereas 36 (35.3%) participants with abdominal attacks experienced 91 TEAEs. No difficulty swallowing sebetralstat was reported. Median (IQR) time to treatment: 11.5 min (1.0-34.0) and 20.0 min (1.0-61.0) for laryngeal and abdominal attacks, respectively; time to beginning of symptom relief: 1.29 h (0.76-3.02) and 1.27 h (0.76-3.54); reduction in attack severity: 4.25 h (1.22 to > 12) and 3.52 h (1.26 to > 12); complete attack resolution: 12.69 h (5.11 to > 24) and 15.17 h (4.46 to > 24). Most mucosal attacks that achieved beginning of symptom relief within 12 h did so with a single dose of sebetralstat (laryngeal: 96.0%; abdominal: 95.8%). Conventional on-demand treatment was administered within 12 h for 3 (9.4%) laryngeal and 43 (8.1%) abdominal attacks.

conclusionOral sebetralstat enabled rapid treatment of laryngeal and abdominal attacks of all severities, was well tolerated, and provided early symptom relief.

Indexed as

angioedemaHAE‐C1INHhereditary angioedemamucosalon‐demand

Identifiers

PMID41252457
PMCPMC12626162

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Registered trials

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.