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

NCT03629184 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: CP405632018-002169-21
• Study Type: interventional
• Target: 173
• Locations: 7 countries
• Sites: 37

Brief 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.

Conditions

Influenza

Outcome Measures

Primary Outcomes (1)

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

  An adverse event (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 serious adverse event (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.

  Up to Day 29

Secondary Outcomes (27)

• Plasma Concentrations of Baloxavir Marboxil - Sparse PK Population

  Days 1 (Post-Dose), 2, 4, 6 and 10

• Plasma Concentrations of S-033447 - Sparse PK Population

  Days 1 (Post-Dose), 2, 4, 6 and 10

• Plasma Concentrations of Baloxavir Marboxil - Extensive PK Population

  Days 1 (Post-Dose), 2, 4, 6 and 10

• Plasma Concentrations of S-033447 - Extensive PK Population

  Days 1 (Post-Dose), 2, 4, 6 and 10

• Time to Alleviation of Influenza Signs and Symptoms

  Up to Day 15

Study Arms (2)

Baloxavir Marboxil

EXPERIMENTAL

Participants will receive a single oral dose of baloxavir marboxil on Day 1 (based on body weight). Oseltamivir matching placebo will also be administered orally twice daily (BID) for 5 days.

Drug: Baloxavir Marboxil

Oseltamivir

ACTIVE COMPARATOR

Participants will receive oseltamivir orally BID for 5 days (based on body weight). Baloxavir marboxil matching placebo will also be administered orally on Day 1

Drug: Oseltamivir

Interventions

Baloxavir Marboxil DRUG

Baloxavir marboxil will be administered as oral suspension in a single dose on Day 1. Oseltamivir matching placebo will also be administered as oral suspension twice daily (BID) for 5 days.

Baloxavir Marboxil

Oseltamivir DRUG

Oseltamivir will be administered as oral suspension BID for 5 days. Participants receiving oseltamivir will also receive baloxavir marboxil matching placebo as oral suspension, single dose on Day 1.

Oseltamivir

Eligibility Criteria

• Age: 1 Year - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (37)

Central Alabama Research; Pediatrics

Birmingham, Alabama, 35209, United States

Location

Harrisburg Family Medical Center

Harrisburg, Arkansas, 72432, United States

Location

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

Jonesboro, Arkansas, 72401, United States

Location

The Probe Medical Research

Los Angeles, California, 90004, United States

Location

Orange County Research Institute

Ontario, California, 91762, United States

Location

Khruz Biotechnology Research Institute

San Diego, California, 92102, United States

Location

Avanza Medical Research Center

Pensacola, Florida, 32503, United States

Location

Clinical Research Prime

Idaho Falls, Idaho, 83404, United States

Location

Cotton O'Neil Clinic; Stormont-Vail Hlth Care

Topeka, Kansas, 66606, United States

Location

Kentucky Pediatric Research Center

Bardstown, Kentucky, 40004, United States

Location

Spiegel, Craig

Bridgeton, Missouri, 63044, United States

Location

Meridian Clinical Research, Llc

Omaha, Nebraska, 68134, United States

Location

Machuca Family Medicine

Las Vegas, Nevada, 89104, United States

Location

OnSite Clinical Solutions LLC

Charlotte, North Carolina, 28203, United States

Location

Montgomery Medical Research /Frontier Clinical Research

Smithfield, North Carolina, 15478, United States

Location

Ohio Pediatric Research Association

Dayton, Ohio, 45414, United States

Location

Coastal Pediatric Research

Charleston, South Carolina, 29414, United States

Location

AFC Urgent Care- Cleveland

Cleveland, Tennessee, 37312, United States

Location

Holston Medical Group

Kingsport, Tennessee, 37660, United States

Location

Oak Cliff Research Company, LLC

Dallas, Texas, 75243, United States

Location

HD Research Corp

Houston, Texas, 77004, United States

Location

Mercury Clinical Research

Houston, Texas, 77036-3316, United States

Location

Tekton Research

San Antonio, Texas, 78240, United States

Location

DM Clinical Research

Tomball, Texas, 77375, United States

Location

ClinPoint Trials

Waxahachie, Texas, 75165, United States

Location

FirstMed East (J Lewis Research)

Salt Lake City, Utah, 84121, United States

Location

Advanced Clinical Research - Jordan Ridge Family Medicine

Salt Lake City, Utah, 84123, United States

Location

ICIMED Instituto de Investigación en Ciencias Médicas

San José, 10108, Costa Rica

Location

Clalit Health Services- Pediatric Ambulatory Clinic; Pediatric Ambulatory Clinic

Petah Tikva, 4931807, Israel

Location

Hospital San Jose; Centro de investigacion y transferencia en salud del Tec de Monterrey

Monterrey, N.L, 64710, Mexico

Location

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

Bydgoszcz, 85-030, Poland

Location

NZOZ Vitamed

Bydgoszcz, 85-079, Poland

Location

Prywatny Gabinet Lekarski

Dębica, 39-200, Poland

Location

NZLA Michalkowice Jarosz i partnerzy Spolka Lekarska

Siemianowice Śląskie, 41-103, Poland

Location

MC Gepatolog

Samara, 443100, Russia

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

Related Publications (1)

• Baker J, Block SL, Matharu B, Burleigh Macutkiewicz L, Wildum S, Dimonaco S, Collinson N, Clinch B, Piedra PA. Baloxavir Marboxil Single-dose Treatment in Influenza-infected Children: A Randomized, Double-blind, Active Controlled Phase 3 Safety and Efficacy Trial (miniSTONE-2). Pediatr Infect Dis J. 2020 Aug;39(8):700-705. doi: 10.1097/INF.0000000000002747.

  PMID: 32516282 DERIVED

MeSH Terms

Conditions

Influenza, Human

Interventions

baloxavir; Oseltamivir

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Acetamides; Amides; Organic Chemicals; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Hydrocarbons

Results Point of Contact

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

genentech@druginfo.com

800 821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 10, 2018
• First Posted: August 14, 2018
• Study Start: November 20, 2018
• Primary Completion: April 3, 2019
• Study Completion: April 3, 2019
• Last Updated: April 29, 2020
• Results First Posted: April 29, 2020

Record last verified: 2020-04

Locations

ES (2); RU (1); US (27); PL (4); ME (1); IL (1); CO (1)