Phase 3
Completed N=173
Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants With Influenza-Like Symptoms
Source: ClinicalTrials.gov NCT03629184 ↗Enrolled (actual)
173
Serious AEs
0.0%
Results posted
Apr 2020
Primary outcomePrimary: Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 46.1; 53.4; 0; 0 percentage of participants
◆ Published Evidence
Highly cited
115citations · ~19 / year
Baloxavir Marboxil Single-dose Treatment in Influenza-infected Children: A Randomized, Double-blind, Active Controlled Phase 3 Safety and Efficacy Trial (miniSTONE-2).
Summary
This study will evaluate the safety, pharmacokinetics, and efficacy of baloxavir marboxil compared with oseltamivir in a single influenza episode in otherwise healthy pediatric participants (i.e., 1 to <12 years of age) with influenza-like symptoms.
Linked Publications
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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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
46.1; 53.4; 0; 0 | — |
| SECONDARY Plasma Concentrations of Baloxavir Marboxil - Sparse PK Population |
0.000; 0.000; 0.000; 0.073; 0.000; 0.000 | — |
| SECONDARY Plasma Concentrations of S-033447 - Sparse PK Population |
45.700; 45.800; 3.110; 49.084; 42.900; 9.233 | — |
| SECONDARY Plasma Concentrations of Baloxavir Marboxil - Extensive PK Population |
0.000; 0.000; 0.000; 0.000; 0.000; 0.000 | — |
| SECONDARY Plasma Concentrations of S-033447 - Extensive PK Population |
10.768; 49.500; 41.000; 28.933; 2.230; 3.131 | — |
| SECONDARY Time to Alleviation of Influenza Signs and Symptoms |
138.1; 150.0 | — |
| SECONDARY Duration of Fever |
41.2; 46.8 | — |
| SECONDARY Duration of Symptoms |
66.4; 67.9 | — |
| SECONDARY Time to Return to Normal Health and Activity |
116.5; 111.6 | — |
| SECONDARY Frequency of Influenza-Related Complications |
6; 4; 0; 0; 0; 0 | — |
| SECONDARY Percentage of Participants With Influenza-Related Complications |
7.4; 7.0; 0; 0; 0; 0 | — |
| SECONDARY Percentage of Participants Requiring Antibiotics |
4.9; 4.7; 0; 0; 2.5; 4.7 | — |
| SECONDARY Time to Cessation of Viral Shedding by Virus Titer |
24.2; 75.8 | — |
| SECONDARY Time to Cessation of Viral Shedding by RT-PCR |
242.5; 238.9 | — |
| SECONDARY Change From Baseline in Influenza Virus Titer at Day 2, 4, 6, 10, 15, 29 |
4.43; 4.27; -3.59; -1.79; -2.83; -2.63 | — |
| SECONDARY Change From Baseline in the Amount of Virus RNA (RT-PCR) at Day 2, 4, 6, 10, 15, 29 |
6.46; 6.86; -1.74; -1.12; -1.78; -2.21 | — |
| SECONDARY Percentage of Participants With Positive Influenza Virus Titer at Day 2, 4, 6, 10 |
100; 100; 15.6; 75.7; 33.3; 50.0 | — |
| SECONDARY Percentage of Participants Positive by RT-PCR at Day 2, 4, 6, 10, 15, 29 |
100; 100; 95.9; 100; 100; 100 | — |
| SECONDARY Area Under the Curve in Virus Titer |
-863.81; -849.29 | — |
| SECONDARY Area Under the Curve in the Amount of Virus RNA (RT-PCR) |
-381.53; -353.31 | — |
| SECONDARY Area Under the Concentration to Time Curve From Time 0 to Infinity (AUC0-inf) of Baloxavir Marboxil |
NA; NA; NA | — |
| SECONDARY Area Under the Concentration to Time Curve From Time 0 to Infinity (AUC0-inf) of S-033447. |
4050; 6600; 4390 | — |
| SECONDARY Maximum Plasma Concentration (Cmax) of Baloxavir Marboxil |
NA; NA; NA | — |
| SECONDARY Maximum Plasma Concentration (Cmax) of S-033447 |
109; 110; 83.2 | — |
| SECONDARY Time to Maximum Plasma Concentration (Tmax) of Baloxavir Marboxil |
NA; NA; NA | — |
| SECONDARY Time to Maximum Plasma Concentration (Tmax) of S-033447 |
4.12; 5.50; 5.55 | — |
| SECONDARY Plasma Concentrations of Baloxavir Marboxil by Dosage |
NA; NA; NA; NA; NA; NA | — |
| SECONDARY Plasma Concentrations of S-033447 by Dosage |
55.7; 75.2; 53.2; 13.2; 28.9; 17.90 | — |
Eligibility Criteria
Inclusion Criteria
- Aged 1 to < 12 years at randomization (Day 1).
- Written informed consent/assent for study participation obtained from participant's parents or legal guardian, with assent as appropriate by the participant, depending on the patient's level of understanding
- Participant able to comply with study requirements, depending on the patient's level of understanding
- Participant with a diagnosis of influenza virus infection confirmed by the presence of all of the following:
- Fever ≥ 38 degree celsius (tympanic temperature) at screening
- At least one respiratory symptom (either cough or nasal congestion)
- The time interval between the onset of symptoms and screening is ≤ 48 hours
Exclusion Criteria
- Severe symptoms of influenza virus infection requiring inpatient treatment
- Concurrent infections requiring systemic antiviral therapy at screening
- Require, in the opinion of the investigator, any of the prohibited medication during the study
- Previous treatment with peramivir, laninamivir, oseltamivir, zanamivir, or amantadine within 2 weeks prior to screening
- Immunization with a live/attenuated influenza vaccine in the 2 weeks prior to randomization
- Concomitant treatment with steroids or other immuno-suppressant therapy
- Known HIV infection or other immunosuppressive disorder
- Uncontrolled renal, vascular, neurologic, or metabolic disease (e.g., diabetes, thyroid disorders, adrenal disease), hepatitis, cirrhosis, or pulmonary disease or participants with known chronic renal failure.
- Active cancer at any site
- History of organ transplantation
- Known allergy to either study drug (i.e., baloxavir marboxil and oseltamivir) or to acetaminophen
- Females with child-bearing potential
- Participation in a clinical trial within 4 weeks or five half-lives of exposure to an investigational drug prior to screening, whichever is longer
Data sourced from ClinicalTrials.gov (NCT03629184) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.