Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants From Birth to < 1 Year With Influenza-Like Symptoms

NCT03653364 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: CP405592018-002154-70
• Study Type: interventional
• Target: 49
• Locations: 9 countries
• Sites: 27

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

Conditions

Influenza

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product. A SAE is any significant hazard, contraindication, side effect that is fatal or life-threatening, requires hospitalization or prolongation of an existing hospitalization, results in persistent or significant disability/ incapacity, is a congenital anomaly/ birth defect, is medically significant or requires intervention to prevent one or other of the outcomes listed above.

  From Day 1 up to Day 29

Secondary Outcomes (19)

• Plasma Concentrations of Baloxavir Marboxil and S-033447

  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

• Area Under the Concentration to Time Curve From Time 0 to Infinity (AUC0-inf) of Baloxavir Marboxil and S-033447

  Up to Day 10

• Maximum Plasma Concentration (Cmax) of Baloxavir Marboxil and S-033447

  Up to Day 10

• Time to Maximum Plasma Concentration (Tmax) of Baloxavir Marboxil and S-033447

  Up to Day 10

• Apparent Half-Life (T1/2) of Baloxavir Marboxil and S-033447

  Up to Day 10

Study Arms (1)

Baloxavir Marboxil

EXPERIMENTAL

Participants will receive single oral dose of baloxavir marboxil on Day 1 (based on body weight and age).

Drug: Baloxavir Marboxil

Interventions

Baloxavir Marboxil DRUG

Participants will receive single oral dose of baloxavir marboxil on Day 1 based on body weight and age. Participants aged ≥ 3 months to \<12 months old received baloxavir marboxil, 2 milligrams per kilograms (mg/kg). Participants from birth to \< 4 weeks old and ≥ 4 weeks to \< 3 months old received baloxavir marboxil, 1 mg/kg.

Baloxavir Marboxil

Eligibility Criteria

• Age: Up to 1 Year
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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:
• In the investigator's judgement there is a clinical suspicion of influenza
• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (27)

The Children's Clinic of Jonesboro, P.A.

Jonesboro, Arkansas, 72401, United States

Location

Usf Health

Tampa, Florida, 33606, United States

Location

Clinical Research Prime

Idaho Falls, Idaho, 83404, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago;Division of Infectious Disease

Chicago, Illinois, 60611, United States

Location

Kentucky Pediatric Research Center

Bardstown, Kentucky, 40004, United States

Location

Oak Cliff Research Company, LLC

Dallas, Texas, 75218, United States

Location

Mercury Clinical Research

Houston, Texas, 77036-3316, United States

Location

Tekton Research

San Antonio, Texas, 78229, United States

Location

MHAT Stamen Iliev AD

Montana, 3400, Bulgaria

Location

SHAT for Pneumo-Phtysiatric Diseases ?Dr. Dimitar Gramatikov?; Dept. of Pulmonology and Phtisiatrics

Rousse, 7002, Bulgaria

Location

ICIMED Instituto de Investigación en Ciencias Médicas

San José, 10108, Costa Rica

Location

TAYS Lastenklinikka; Lasten lääketutkimuskeskus Peetu

Tampere, 33520, Finland

Location

Clalit Health Services- Pediatric Ambulatory Clinic; Pediatric Ambulatory Clinic

Petah Tikva, 4931807, Israel

Location

NZOZ Salmed

??czna, 21-010, Poland

Location

Samodzielny Zespol Publicznych Zakladow Opieki Zdrowotnej im. Dzieci Warszawy w Dziekanowie Lesnym

?omianki, 05-092, Poland

Location

Wojewodzki Szpital Obserwacyjno-Zakazny; Oddzia? Pediatrii, Chorób Infekcyjnych i Hepatologii

Bydgoszcz, 85-030, Poland

Location

IN VIVO Sp. z o.o.

Bydgoszcz, 85-048, Poland

Location

NZOZ Vitamed

Bydgoszcz, 85-079, Poland

Location

The scientific-research institute of epidemiology; Infectious clinical hospital ?2 of Moscow H.D.

Moskva, Moscow Oblast, 111123, Russia

Location

Global Clinical Trials; Clinical Trials

Gauteng, 0001, South Africa

Location

GAMA; Clinical Research

Germiston, 1401, South Africa

Location

Peermed Clinical Trial Centre

Kempton Park, 1619, South Africa

Location

Metropolitan Clinical Research Institute

Polokwane, 0699, South Africa

Location

Pholosho Netcare; Netcare Hospital

Polokwane, 0699, South Africa

Location

Setshaba Research Centre; Clinical Research

Pretoria, 0152, South Africa

Location

Complejo Hospitalario Universitario de Santiago (CHUS); Area Asistencial Integrada de Pediatría

Santiago de Compostela, LA Coruña, 15706, Spain

Location

Hospital Universitario 12 de Octubre; Servicio de Pediatria

Madrid, 28041, Spain

Location

MeSH Terms

Conditions

Influenza, Human

Interventions

baloxavir

Condition Hierarchy (Ancestors)

Respiratory Tract Infections; Infections; Orthomyxoviridae Infections; RNA Virus Infections; Virus Diseases; Respiratory Tract Diseases

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

800 821-8590

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 29, 2018
• First Posted: August 31, 2018
• Study Start: January 23, 2019
• Primary Completion: April 3, 2023
• Study Completion: April 3, 2023
• Last Updated: May 17, 2024
• Results First Posted: May 17, 2024

Record last verified: 2024-05

Locations

BU (2); RU (1); PL (5); US (8); CO (1); ES (2); ZA (6); IL (1); FI (1)