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

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ClinicalTrials.gov Identifier: NCT03653364
Recruitment Status : Not yet recruiting
First Posted : August 31, 2018
Last Update Posted : August 31, 2018
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

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

Condition or disease Intervention/treatment Phase
Influenza Drug: Baloxavir Marboxil Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Study Start Date : October 27, 2018
Estimated Primary Completion Date : May 23, 2020
Estimated Study Completion Date : May 23, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Flu Flu Shot

Arm Intervention/treatment
Experimental: Baloxavir Marboxil
Participants will receive single oral dose of baloxavir marboxil on Day 1 (based on body weight and age).
Drug: Baloxavir Marboxil

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

Participants will be recruited in parallel to the following three cohorts:

Cohort I: ≥ 3 months to < 12 months old (minimum 8 participants): 2 mg/kg

Cohort II: ≥ 4 weeks to < 3 months old (minimum 4 patients): 1 mg/kg

Cohort III: birth to < 4 weeks old (minimum 4 patients): 1 mg/kg





Primary Outcome Measures :
  1. Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to Day 29 ]
    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.


Secondary Outcome Measures :
  1. Plasma Concentrations of Baloxavir Marboxil and S-033447 [ Time Frame: Day 2 and Day 4 ]
  2. Area Under the Concentration to Time Curve from Time 0 to Infinity (AUC0-inf) of baloxavir marboxil and S-033447 [ Time Frame: Up to Day 10 ]
  3. Maximum Plasma Concentration (Cmax) of baloxavir marboxil and S-033447 [ Time Frame: Up to Day 10 ]
  4. Time to Maximum Plasma Concentration (Tmax) of baloxavir marboxil and S-033447 [ Time Frame: Up to Day 10 ]
  5. Apparent Half-Life (T1/2) of baloxavir marboxil and S-033447 [ Time Frame: Up to Day 10 ]
  6. Time to Alleviation of Influenza Signs and Symptoms [ Time Frame: Up to Day 15 ]

    Time to alleviation of influenza signs and symptoms is defined as the length of time taken from the start of treatment to the point at which all of the following criteria are met and remain so for at least 21.5 hours:

    • A score of 0 (no problem) or 1 (minor problem) for cough and nasal symptoms (items 14 and 15 of the Canadian Acute Respiratory Illness and Flu Scale [CARIFS])
    • A "yes" response to the following question on the CARIFS: "Since the last assessment has the subject been able to return to day care/school, or resume his or her normal daily activity in the same way as performed prior to developing the flu?"
    • First return to afebrile state (tympanic temperature ≤37.2 degree Celsius [°C])

  7. Duration of Fever [ Time Frame: Up to Day 15 ]
    Length of time taken by participants to return to afebrile state [tympanic temperature ≤ 37.2°C] and remaining so for at least 21.5 hours.

  8. Duration of Symptoms [ Time Frame: Up to Day 15 ]
    The efficacy of baloxavir marboxil is evaluated by duration of symptoms i.e., alleviation of all symptoms as defined by a score of 0 [no problem] or 1 [minor problem] and remaining so for at least 21.5 hours, for all 18 symptoms specified in the CARIFS questionnaire).

  9. Time to Return to Normal Health and Activity [ Time Frame: Up to Day 15 ]
  10. Frequency of Influenza-Related Complications [ Time Frame: Up to Day 29 ]
    The influenza related complications include death, hospitalization, radiologically confirmed pneumonia, bronchitis, sinusitis, otitis media, encephalitis/encephalopathy, febrile seizures, myositis.

  11. Percentage of Participants Requiring Antibiotics [ Time Frame: Up to Day 29 ]
  12. Time to Cessation of Viral Shedding by Virus Titer and by RT-PCR [ Time Frame: Day 1 - Day 29 ]
  13. Change from Baseline in Influenza Virus Titer and in the Amount of Virus RNA (RT-PCR) at Day 2, 4, 6, 10, 15, 29 [ Time Frame: Baseline, Day 2, 4, 6, 10, 15, 29 ]
  14. Percentage of Participants with Positive Influenza Virus Titer and Positive by RT-PCR at Day 2, 4, 6, 10, 15, 29 [ Time Frame: Day 2, 4, 6, 10, 15, 29 ]
  15. Area Under the Curve in Virus Titer and in the Amount of Virus RNA (RT-PCR) [ Time Frame: Day 1 - Day 29 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 1 Year   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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:
  • Fever ≥ 38°C (tympanic temperature) at screening
  • At least one respiratory symptom (either cough or coryza)
  • 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 allergy 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03653364


Contacts
Contact: Reference Study ID Number: CP40559 www.roche.com/about_roche/roche_worldwide.htm 888-662-6728 (U.S. and Canada) global-roche-genentech-trials@gene.com

Locations
United States, Alabama
Uni of Alabama At Birmingham; Departement of Medicine Not yet recruiting
Birmingham, Alabama, United States, 35233
United States, Arkansas
The Children's Clinic of Jonesboro, P.A. Not yet recruiting
Jonesboro, Arkansas, United States, 72401
United States, Colorado
University of Colorado Denver, Children's Hospital Not yet recruiting
Aurora, Colorado, United States, 80045
United States, Florida
Avanza Medical Research Center Not yet recruiting
Pensacola, Florida, United States, 32503
USF Tampa General Hospital Not yet recruiting
Tampa, Florida, United States, 33606
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago;Division of Infectious Disease Not yet recruiting
Chicago, Illinois, United States, 60611
United States, Kentucky
Kentucky Pediatric Research Center Not yet recruiting
Bardstown, Kentucky, United States, 40004
United States, Minnesota
Children's Minnesota Not yet recruiting
Minneapolis, Minnesota, United States, 55404
United States, Nebraska
Midwest Childrens Health Research Institute Not yet recruiting
Lincoln, Nebraska, United States, 68516
United States, New York
SUNY Upstate Medical University Not yet recruiting
Syracuse, New York, United States, 13210
United States, South Carolina
Coastal Pediatric Research Not yet recruiting
Charleston, South Carolina, United States, 29414
United States, Texas
Oak Cliff Research Company, LLC Not yet recruiting
Dallas, Texas, United States, 75231
DM Clinical Research Not yet recruiting
Tomball, Texas, United States, 77375
Israel
Clalit Health Services- Pediatric Ambulatory Clinic; Pediatric Ambulatory Clinic Not yet recruiting
Petach Tikva, Israel
South Africa
LCS Clinical Research Unit; Clinical Research Not yet recruiting
Cosmo CITY, South Africa, 2188
CRISMO; Clinical Research Not yet recruiting
Germiston, South Africa, 1401
Peermed Clinical Trial Centre Not yet recruiting
Kempton Park, South Africa, 1619
Setshaba Research Centre; Clinical Research Not yet recruiting
Pretoria, South Africa, 0152
Clinical Projects Research SA pty Ltd Not yet recruiting
Worcester, South Africa, 6850
Sponsors and Collaborators
Hoffmann-La Roche

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03653364     History of Changes
Other Study ID Numbers: CP40559
2018-002154-70 ( EudraCT Number )
First Posted: August 31, 2018    Key Record Dates
Last Update Posted: August 31, 2018
Last Verified: August 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Influenza, Human
Orthomyxoviridae Infections
RNA Virus Infections
Virus Diseases
Respiratory Tract Infections
Respiratory Tract Diseases