A Study to Assess the Safety, Pharmacokinetics (how the body affects the drug), and Efficacy (how well the drug works) of Baloxavir Marboxil in Otherwise Healthy Pediatric Patients from Birth to < 1 Year with Influenza-Like Symptoms

Phase 1

• Conditions: Influenza; MedDRA version: 20.0Level: LLTClassification code 10022001Term: Influenza (epidemic)System Organ Class: 100000004862; MedDRA version: 20.1Level: LLTClassification code 10022003Term: Influenza B virus infectionSystem Organ Class: 100000004862; MedDRA version: 20.1Level: LLTClassification code 10022002Term: Influenza A virus infectionSystem Organ Class: 100000004862; MedDRA version: 20.0Level: LLTClassification code 10016790Term: FluSystem Organ Class: 100000004862; Therapeutic area: Diseases [C] - Virus Diseases [C02]

• Registration Number: EUCTR2018-002154-70-FI
• Lead Sponsor: F. Hoffmann-La Roche Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-07-12
• Last Update Posted: 12 December 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

- Age from birth to < 1 year at screening
- Parent/guardian willing and able to comply with study requirements, in the investigator’s judgment
- Patients with a diagnosis of influenza virus infection confirmed by the presence of all of the following:
o In the investigator's judgement there is a clinical suspicion of
influenza
o At least one respiratory symptom (either cough or coryza)
o Positive prescreening influenza test (Rapid Influenza Diagnostic Test or polymerase chain reaction [PCR]) performed within 48 hours of screening
Results from local testing as part of standard of care are acceptable if
samples are collected within 48 hours of screening and recorded in the
patients' medical records.
-Patients with a negative prescreening COVID-19 test (RAT or PCR) performed within 48 hours of screening
Results from local testing as part of standard of care are acceptable if
samples are collected within 48 hours of screening and recorded in the
patients' medical records
-The time interval between the onset of symptoms and screening is <= 96 hours

Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Hospitalized for complications of influenza or significant comorbidities
o Patients hospitalized for observation, monitoring or precautionary
supportive care may still be acceptable for enrollment provided all
eligibility criteria are met.
- Concurrent infections requiring systemic antiviral therapy at screening
- Require, in the opinion of the investigator, any of the prohibited medication during the study
- Preterm neonates (born at < 37 weeks gestation)
- Weigh < 2.5 kg at screening
o Only children > 4.5 kg will be eligible to take part in countries that have volume limits for blood draws set at 1% of the total blood volume (maximum over 24 hour period) and 3% of the total blood volume (maximum over 30-day period)
- Previous treatment with peramivir, laninamivir, oseltamivir, zanamivir, or amantadine within 2 weeks prior to screening
- Immunization with a live/attenuated influenza vaccine during the 2 weeks prior to screening
- Concomitant treatment with steroids or other immuno-suppressant therapy
- Known HIV infection or other immunosuppressive disorder
- Uncontrolled renal, vascular, neurologic or metabolic disease, hepatitis, cirrhosis, or pulmonary disease or patients with known chronic renal failure
- Active cancer at any site
- History of organ transplant
- Known hypersensitivity to study drug (i.e. baloxavir marboxil) or the drug excipients
- Known hypersensitivity to acetaminophen
- Participation in a clinical trial within 4 weeks or five half-lives of exposure to an investigational drug prior to screening, whichever is longer

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: • To evaluate the safety of a single dose of baloxavir marboxil<br>;Secondary Objective: • To evaluate the efficacy of baloxavir marboxil<br>• To evaluate the virological activity of baloxavir marboxil<br>• To evaluate the pharmacokinetics (PK) of baloxavir marboxil after single dose administration;Primary end point(s): 1. Incidence, severity, and timing of adverse events, serious adverse events, vital sign measurements, and clinical laboratory tests<br>;Timepoint(s) of evaluation of this end point: 1. Up to Day 29 or early termination<br>