Clinical trial NCT03653364: Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants From Birth to < 1 Year With Influenza-Like Symptoms
Official title: A Multicenter, Single-Arm, Open-Label Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Otherwise Healthy Pediatric Patients From Birth to < 1 Year With Influenza-Like Symptoms
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Influenza
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2018-08-31
Enrollment: 49
Sex: ALL
Maximum age (years): 1
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will evaluate the safety, pharmacokinetics and efficacy of baloxavir marboxil in healthy pediatric participants from birth to \<1 year with influenza like symptoms


Eligibility criteria: Inclusion Criteria:

* Age from birth to \< 1 year at screening
* Written informed consent for study participation obtained from participant's parents or legal guardian
* Parent/guardian willing and able to comply with study requirements, in the investigator's judgment
* Participants with a diagnosis of influenza virus infection confirmed by the presence of all of the following:

  1. In the investigator's judgement there is a clinical suspicion of influenza
  2. At least one respiratory symptom (either cough or coryza)

  (b) Positive prescreening influenza test (RIDT or PCR) performed within 48 hours of screening
* Participants with a negative prescreening COVID-19 test (RAT or PCR) within 48 hours of screening
* The time interval between the onset of symptoms and screening is ≤ 96 hours (the onset of symptoms is defined as the time when body temperature first exceeded 37.5°C if known, or the time when the first symptom was noticed by the parent or caregiver)

Exclusion Criteria:

* Hospitalized for complications of influenza or significant comorbidities
* 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) and/or weighing \< 2.5 kg at screening
* 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 (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 transplant
* Known hypersensitivity to study drug (i.e., baloxavir marboxil) or 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

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From Day 1 up to Day 29
Secondary outcomes:
measure: Plasma Concentrations of Baloxavir Marboxil and S-033447
timeFrame: 0.5 to 2 hours post dose on Day 1; 24 hours (Day 2) and 72 hours (Day 4) post dose, Day 6 and Day 10
measure: Area Under the Concentration to Time Curve From Time 0 to Infinity (AUC0-inf) of Baloxavir Marboxil and S-033447
timeFrame: Up to Day 10
measure: Maximum Plasma Concentration (Cmax) of Baloxavir Marboxil and S-033447
timeFrame: Up to Day 10
measure: Time to Maximum Plasma Concentration (Tmax) of Baloxavir Marboxil and S-033447
timeFrame: Up to Day 10
measure: Apparent Half-Life (T1/2) of Baloxavir Marboxil and S-033447
timeFrame: Up to Day 10
measure: Time to Alleviation of Influenza Signs and Symptoms
timeFrame: Day 1 up to Day 15
measure: Duration of Fever
timeFrame: Day 1 up to Day 15
measure: Duration of Symptoms
timeFrame: Day 1 up to Day 15
measure: Time to Return to Normal Health and Activity
timeFrame: Day 1 up to Day 15
measure: Number of Participants With Influenza-Related Complications
timeFrame: Day 1 up to Day 29
measure: Number of Participants Requiring Antibiotics
timeFrame: Day 1 up to Day 29
measure: Time to Cessation of Viral Shedding by Virus Titer
timeFrame: Day 1 up to Day 29
measure: Time to Cessation of Viral Shedding by Reverse Transcription-Polymerase Chain Reaction (RT-PCR)
timeFrame: Day 1 up to Day 29
measure: Change From Baseline in Influenza Virus Titer Over Time
timeFrame: Baseline, Days 2, 4, 6, 10, and 29
measure: Change From Baseline in the Amount of Virus RNA (RT-PCR) Over Time
timeFrame: Baseline, Days 2, 4, 6, and 10
measure: Percentage of Participants With Positive Influenza Virus Titer Over Time
timeFrame: Baseline, Days 2, 4, 6, 10, and 29
measure: Percentage of Participants Positive by RT-PCR Over Time
timeFrame: Baseline, Days 2, 4, 6, 10, and 29
measure: Area Under the Concentration-Time Curve (AUC) in Virus Titer
timeFrame: Day 1 up to Day 29
measure: Area Under the Curve in the Amount of Virus RNA (RT-PCR)
timeFrame: Day 1 up to Day 29
Other outcomes:
Locations:
city: Jonesboro
country: United States
facility: The Children's Clinic of Jonesboro, P.A.
state: Arkansas
city: Tampa
country: United States
facility: Usf Health
state: Florida
city: Idaho Falls
country: United States
facility: Clinical Research Prime
state: Idaho
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago;Division of Infectious Disease
state: Illinois
city: Bardstown
country: United States
facility: Kentucky Pediatric Research Center
state: Kentucky
city: Dallas
country: United States
facility: Oak Cliff Research Company, LLC
state: Texas
city: Houston
country: United States
facility: Mercury Clinical Research
state: Texas
city: San Antonio
country: United States
facility: Tekton Research
state: Texas
city: Montana
country: Bulgaria
facility: MHAT Stamen Iliev AD
city: Ruse
country: Bulgaria
facility: SHAT for Pneumo-Phtysiatric Diseases ?Dr. Dimitar Gramatikov?; Dept. of Pulmonology and Phtisiatrics
city: San José
country: Costa Rica
facility: ICIMED Instituto de Investigación en Ciencias Médicas
city: Tampere
country: Finland
facility: TAYS Lastenklinikka; Lasten lääketutkimuskeskus Peetu
city: Petach Tikva
country: Israel
facility: Clalit Health Services- Pediatric Ambulatory Clinic; Pediatric Ambulatory Clinic
city: ??czna
country: Poland
facility: NZOZ Salmed
city: ?omianki
country: Poland
facility: Samodzielny Zespol Publicznych Zakladow Opieki Zdrowotnej im. Dzieci Warszawy w Dziekanowie Lesnym
city: Bydgoszcz
country: Poland
facility: Wojewodzki Szpital Obserwacyjno-Zakazny; Oddzia? Pediatrii, Chorób Infekcyjnych i Hepatologii
city: Bydgoszcz
country: Poland
facility: IN VIVO Sp. z o.o.
city: Bydgoszcz
country: Poland
facility: NZOZ Vitamed
city: Moskva
country: Russian Federation
facility: The scientific-research institute of epidemiology; Infectious clinical hospital ?2 of Moscow H.D.
state: Moskovskaja Oblast
city: Gauteng
country: South Africa
facility: Global Clinical Trials; Clinical Trials
city: Germiston
country: South Africa
facility: GAMA; Clinical Research
city: Kempton Park
country: South Africa
facility: Peermed Clinical Trial Centre
city: Polokwane
country: South Africa
facility: Metropolitan Clinical Research Institute
city: Polokwane
country: South Africa
facility: Pholosho Netcare; Netcare Hospital
city: Pretoria
country: South Africa
facility: Setshaba Research Centre; Clinical Research
city: Santiago de Compostela
country: Spain
facility: Complejo Hospitalario Universitario de Santiago (CHUS); Area Asistencial Integrada de Pediatría
state: LA Coruña
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre; Servicio de Pediatria
Study officials:
References: