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Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: A randomized, double-blind, placebo-controlled phase 3 trial
B. Zuraw, WR. Lumry, DT. Johnston, E. Aygören-Pürsün, A. Banerji, JA. Bernstein, SC. Christiansen, JS. Jacobs, KV. Sitz, RG. Gower, R. Gagnon, HJ. Wedner, T. Kinaciyan, R. Hakl, J. Hanzlíková, JT. Anderson, DL. McNeil, SB. Fritz, WH. Yang, R....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu klinické zkoušky, fáze III, časopisecké články, randomizované kontrolované studie, Research Support, N.I.H., Extramural, práce podpořená grantem
Grantová podpora
UL1 TR001102
NCATS NIH HHS - United States
- MeSH
- aplikace orální MeSH
- dospělí MeSH
- dvojitá slepá metoda MeSH
- hereditární angioedémy farmakoterapie MeSH
- lidé MeSH
- plazmatický kalikrein aplikace a dávkování MeSH
- prospektivní studie MeSH
- pyrazoly aplikace a dávkování MeSH
- výsledek terapie MeSH
- Check Tag
- dospělí MeSH
- lidé MeSH
- mužské pohlaví MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
- klinické zkoušky, fáze III MeSH
- práce podpořená grantem MeSH
- randomizované kontrolované studie MeSH
- Research Support, N.I.H., Extramural MeSH
BACKGROUND: Berotralstat (BCX7353) is an oral, once-daily inhibitor of plasma kallikrein in development for the prophylaxis of hereditary angioedema (HAE) attacks. OBJECTIVE: Our aim was to determine the efficacy, safety, and tolerability of berotralstat in patients with HAE over a 24-week treatment period (the phase 3 APeX-2 trial). METHODS: APeX-2 was a double-blind, parallel-group study that randomized patients at 40 sites in 11 countries 1:1:1 to receive once-daily berotralstat in a dose of 110 mg or 150 mg or placebo (Clinicaltrials.gov identifier NCT03485911). Patients aged 12 years or older with HAE due to C1 inhibitor deficiency and at least 2 investigator-confirmed HAE attacks in the first 56 days of a prospective run-in period were eligible. The primary efficacy end point was the rate of investigator-confirmed HAE attacks during the 24-week treatment period. RESULTS: A total of 121 patients were randomized; 120 of them received at least 1 dose of the study drug (n = 41, 40, and 39 in the 110-mg dose of berotralstat, 150-mg of dose berotralstat, and placebo groups, respectively). Berotralstat demonstrated a significant reduction in attack rate at both 110 mg (1.65 attacks per month; P = .024) and 150 mg (1.31 attacks per month; P < .001) relative to placebo (2.35 attacks per month). The most frequent treatment-emergent adverse events that occurred more with berotralstat than with placebo were abdominal pain, vomiting, diarrhea, and back pain. No drug-related serious treatment-emergent adverse events occurred. CONCLUSION: Both the 110-mg and 150-mg doses of berotralstat reduced HAE attack rates compared with placebo and were safe and generally well tolerated. The most favorable benefit-to-risk profile was observed at a dose of 150 mg per day.
Allergy and Asthma Clinical Research Walnut Creek Calif
Allergy and Asthma Specialists of Dallas Dallas Tex
Asthma and Allergy Specialists Charlotte NC
BioCryst Pharmaceuticals Durham NC
Clinical Research Center of Alabama Birmingham Ala
Clinique Spécialisée en Allergie de la Capitale Québec Canada
Department of Medicine and Pediatrics Penn State University Hershey Pa
Department of Pediatrics University of California Los Angeles Calif
Faculty Hospital Department of Allergology and Immunology Plzen Czech Republic
Institute for Asthma and Allergy Chevy Chase Md
Little Rock Allergy and Asthma Clinical Research Center Little Rock Ark
Massachusetts General Hospital Harvard Medical School Boston Mass
Medical University of Vienna Department of Dermatology Vienna Austria
Optimed Research Ltd Columbus Ohio
Portland Clinical Research Portland Ore
University Hospital Frankfurt Goethe University Frankfurt Frankfurt Germany
University of California San Diego San Diego Calif
University of Cincinnati Cincinnati Ohio
University of Washington School of Medicine Marycliff Clinical Research Spokane Wash
Citace poskytuje Crossref.org
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