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Baloxavir Marboxil Single-dose Treatment in Influenza-infected Children: A Randomized, Double-blind, Active Controlled Phase 3 Safety and Efficacy Trial (miniSTONE-2).
Pediatr Infect Dis J. 2020 08; 39(8):700-705.PI

Abstract

BACKGROUND

Baloxavir marboxil (baloxavir) is a novel, cap-dependent endonuclease inhibitor that has previously demonstrated efficacy in the treatment of influenza in adults and adolescents. We assessed the safety and efficacy of baloxavir in otherwise healthy children with acute influenza.

METHODS

MiniSTONE-2 (Clinicaltrials.gov: NCT03629184) was a double-blind, randomized, active controlled trial enrolling children 1-<12 years old with a clinical diagnosis of influenza. Children were randomized 2:1 to receive either a single dose of oral baloxavir or oral oseltamivir twice daily for 5 days. The primary endpoint was incidence, severity and timing of adverse events (AEs); efficacy was a secondary endpoint.

RESULTS

In total, 173 children were randomized and dosed, 115 to the baloxavir group and 58 to the oseltamivir group. Characteristics of participants were similar between treatment groups. Overall, 122 AEs were reported in 84 (48.6%) children. Incidence of AEs was similar between baloxavir and oseltamivir groups (46.1% vs. 53.4%, respectively). The most common AEs were gastrointestinal (vomiting/diarrhea) in both groups [baloxavir: 12 children (10.4%); oseltamivir: 10 children (17.2%)]. No deaths, serious AEs or hospitalizations were reported. Median time (95% confidence interval) to alleviation of signs and symptoms of influenza was similar between groups: 138.1 (116.6-163.2) hours with baloxavir versus 150.0 (115.0-165.7) hours with oseltamivir.

CONCLUSIONS

Oral baloxavir is well tolerated and effective at alleviating symptoms in otherwise healthy children with acute influenza. Baloxavir provides a new therapeutic option with a simple oral dosing regimen.

Authors+Show Affiliations

From the Clinical Research Prime, Idaho Falls, Idaho.Kentucky Pediatric and Adult Research Inc., Bardstown, Kentucky.F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.F. Hoffmann-La Roche Ltd, Basel, Switzerland.F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.F. Hoffmann-La Roche Ltd, Welwyn Garden City, Hertfordshire, United Kingdom.Baylor College of Medicine, Houston, Texas.

Pub Type(s)

Clinical Trial, Phase III
Journal Article
Multicenter Study
Randomized Controlled Trial
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

32516282

Citation

Baker, Jeffrey, et al. "Baloxavir Marboxil Single-dose Treatment in Influenza-infected Children: a Randomized, Double-blind, Active Controlled Phase 3 Safety and Efficacy Trial (miniSTONE-2)." The Pediatric Infectious Disease Journal, vol. 39, no. 8, 2020, pp. 700-705.
Baker J, Block SL, Matharu B, et al. Baloxavir Marboxil Single-dose Treatment in Influenza-infected Children: A Randomized, Double-blind, Active Controlled Phase 3 Safety and Efficacy Trial (miniSTONE-2). Pediatr Infect Dis J. 2020;39(8):700-705.
Baker, J., Block, S. L., Matharu, B., Burleigh Macutkiewicz, L., Wildum, S., Dimonaco, S., Collinson, N., Clinch, B., & Piedra, P. A. (2020). Baloxavir Marboxil Single-dose Treatment in Influenza-infected Children: A Randomized, Double-blind, Active Controlled Phase 3 Safety and Efficacy Trial (miniSTONE-2). The Pediatric Infectious Disease Journal, 39(8), 700-705. https://doi.org/10.1097/INF.0000000000002747
Baker J, et al. Baloxavir Marboxil Single-dose Treatment in Influenza-infected Children: a Randomized, Double-blind, Active Controlled Phase 3 Safety and Efficacy Trial (miniSTONE-2). Pediatr Infect Dis J. 2020;39(8):700-705. PubMed PMID: 32516282.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Baloxavir Marboxil Single-dose Treatment in Influenza-infected Children: A Randomized, Double-blind, Active Controlled Phase 3 Safety and Efficacy Trial (miniSTONE-2). AU - Baker,Jeffrey, AU - Block,Stanley L, AU - Matharu,Balpreet, AU - Burleigh Macutkiewicz,Laura, AU - Wildum,Steffen, AU - Dimonaco,Sophie, AU - Collinson,Neil, AU - Clinch,Barry, AU - Piedra,Pedro A, PY - 2020/6/10/pubmed PY - 2021/7/16/medline PY - 2020/6/10/entrez SP - 700 EP - 705 JF - The Pediatric infectious disease journal JO - Pediatr Infect Dis J VL - 39 IS - 8 N2 - BACKGROUND: Baloxavir marboxil (baloxavir) is a novel, cap-dependent endonuclease inhibitor that has previously demonstrated efficacy in the treatment of influenza in adults and adolescents. We assessed the safety and efficacy of baloxavir in otherwise healthy children with acute influenza. METHODS: MiniSTONE-2 (Clinicaltrials.gov: NCT03629184) was a double-blind, randomized, active controlled trial enrolling children 1-<12 years old with a clinical diagnosis of influenza. Children were randomized 2:1 to receive either a single dose of oral baloxavir or oral oseltamivir twice daily for 5 days. The primary endpoint was incidence, severity and timing of adverse events (AEs); efficacy was a secondary endpoint. RESULTS: In total, 173 children were randomized and dosed, 115 to the baloxavir group and 58 to the oseltamivir group. Characteristics of participants were similar between treatment groups. Overall, 122 AEs were reported in 84 (48.6%) children. Incidence of AEs was similar between baloxavir and oseltamivir groups (46.1% vs. 53.4%, respectively). The most common AEs were gastrointestinal (vomiting/diarrhea) in both groups [baloxavir: 12 children (10.4%); oseltamivir: 10 children (17.2%)]. No deaths, serious AEs or hospitalizations were reported. Median time (95% confidence interval) to alleviation of signs and symptoms of influenza was similar between groups: 138.1 (116.6-163.2) hours with baloxavir versus 150.0 (115.0-165.7) hours with oseltamivir. CONCLUSIONS: Oral baloxavir is well tolerated and effective at alleviating symptoms in otherwise healthy children with acute influenza. Baloxavir provides a new therapeutic option with a simple oral dosing regimen. SN - 1532-0987 UR - https://www.unboundmedicine.com/medline/citation/32516282/Baloxavir_Marboxil_Single_dose_Treatment_in_Influenza_infected_Children:_A_Randomized_Double_blind_Active_Controlled_Phase_3_Safety_and_Efficacy_Trial__miniSTONE_2__ DB - PRIME DP - Unbound Medicine ER -