臨床研究等提出・公開システム

A phase 3 randomized, double-blind, placebo-controlled study to confirm the efficacy of a single dose of baloxavir marboxil in the prevention of influenza virus infection

Study to confirm the efficacy of baloxavir marboxil versus placebo in the prevention of influenza virus infection

752

In the mITT population, the majority of subjects were female (baloxavir marboxil: 79.4% and placebo: 77.3%), parents of index patients (71.4% and 67.2%). The mean age was 33.5 and 33.6 years, the proportion of subjects < 12 years of age was 19.0% and 18.9% and the proportion of subjects >= 65 years of age was 2.1% and 4.0% in the baloxavir marboxil and placebo groups, respectively. The proportion of subjects with high risk factor was 12.3% and 13.9% in the baloxavir marboxil and placebo groups, respectively. The common influenza virus subtypes based on RT-PCR of patients with influenza virus infection who have a subject included in mITT population were A/H3NX (48.4% and 48.8%), and A/H1N1pdm (47.1% and 48.0%) and B type was reported in few patients (0.5% and 0.8%).

A total of 752 subjects (375 in the baloxavir marboxil group and 377 in the placebo group) were randomized as household members of patients with influenza virus infection. Of these, 373 (99.5%) and 375 (99.5%) subjects completed the study and 2 (0.5%) subjects each were withdrawn from the study in the baloxavir marboxil and placebo groups, respectively.

Only 1 serious adverse event (SAE) and adverse event (AE) leading to withdrawal from the study (psychotic disorder in the placebo group) was reported in this study. No deaths, SAEs, or AEs leading to withdrawal were reported in baloxavir marboxil group.

The proportion of subjects who were infected with influenza virus (RT-PCR positive) and presented with fever and at least one respiratory symptom from Day 1 to Day 10 in the mITT population was significantly lower in the baloxavir marboxil group compared with the placebo group; 1.9% (7/374 subjects) in the baloxavir marboxil group and 13.6% (51/375 subjects) in the placebo group (adjusted risk ratio: 0.14 [95% CI: 0.06, 0.30], p < 0.0001).

The incidences of AEs were 22.2% (83/374 subjects, 102 events) in the baloxavir marboxil group and 20.5% (77/375 subjects, 99 events) in the placebo group. The incidences of treatment-related AEs were 1.9% (7/374 subjects, 7 events) in the baloxavir marboxil group and 1.6% (6/375 subjects, 7 events) in the placebo group. The incidences of AEs and treatment-related AEs were similar between the treatment groups.

The efficacy of baloxavir marboxil was demonstrated compared with placebo in the prevention of influenza virus infection in subjects who were household members of influenza-infected patients. A single oral dose of baloxavir marboxil for the prevention of influenza virus infection was well tolerated.

July. 08, 2020

https://www.nejm.org/doi/full/10.1056/NEJMoa1915341

Yes

Individual patients data from the study will be shared on a portal site (https://clinicalstudydatarequest.com) when it become shareable. Requests from researchers will be reviewed by an Independent Review Panel, consisting of third-party experts, with regard to the validity and feasibility of the research that the researchers intend to carry out. When the request is approved by the panel, the researchers will be requested to execute a contract (which will have certain requirements with respect to personal data privacy, confidentiality, and compliance with the law) with Shionogi before being provided with access to the clinical trial data. Individual patient data will be shared after anonymization in order to protect the privacy of study participants and to comply with laws and regulations (including but not limited to the terms of the patient informed consent forms).

Shionogi & Co., Ltd.

shionogiclintrials-admin@shionogi.co.jp

Shionogi & Co., Ltd.

+81-6-6209-7885

shionogiclintrials-admin@shionogi.co.jp

completed

Nov. 09, 2018

Interventional

Multicenter, randomized, double-blind, parallel-group

prevention purpose

3

-Subjects who had lived with the patients with influenza virus infection for 48 hours or more prior to informed consent.
-Subjects who are judged not to have influenza virus infection by the investigator.
etc.

-Subjects who are unable to live with the index patient from Screening until Day 10.
-Subjects who have any underlying diseases requiring systemic (oral or injection), or nasal treatment of antipyretics/analgesics, corticosteroids, or immunosuppressive agents.
etc.

Both

Influenza A and/or B virus infection

investigational material(s)
Generic name etc : baloxavir marboxil
INN of investigational material : baloxavir marboxil
Therapeutic category code : 625 Anti-virus agents
Dosage and Administration for Investigational material : Oral

control material(s)
Generic name etc : -
INN of investigational material : -
Therapeutic category code :
Dosage and Administration for Investigational material : -

efficacy
Proportion of subjects who are infected with influenza virus (RT-PCR positive), and present with fever and at least one respiratory symptom in the period from Day 1 to Day 10

safety
efficacy
pharmacokinetics
Percentage of patients with any adverse events, etc.

Sept. 20, 2018