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 : Recruiting
First Posted : August 31, 2018
Last Update Posted : February 5, 2020
|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)|
|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|
|Actual Study Start Date :||January 23, 2019|
|Estimated Primary Completion Date :||September 2, 2020|
|Estimated Study Completion Date :||September 2, 2020|
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
- 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.
- Plasma Concentrations of Baloxavir Marboxil and S-033447 [ Time Frame: Day 2 and Day 4 ]
- 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 ]
- Maximum Plasma Concentration (Cmax) of baloxavir marboxil and S-033447 [ Time Frame: Up to Day 10 ]
- Time to Maximum Plasma Concentration (Tmax) of baloxavir marboxil and S-033447 [ Time Frame: Up to Day 10 ]
- Apparent Half-Life (T1/2) of baloxavir marboxil and S-033447 [ Time Frame: Up to Day 10 ]
- 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])
- 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.
- 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).
- Time to Return to Normal Health and Activity [ Time Frame: Up to Day 15 ]
- 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.
- Percentage of Participants Requiring Antibiotics [ Time Frame: Up to Day 29 ]
- Time to Cessation of Viral Shedding by Virus Titer and by RT-PCR [ Time Frame: Day 1 - Day 29 ]
- 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 ]
- 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 ]
- Area Under the Curve in Virus Titer and in the Amount of Virus RNA (RT-PCR) [ Time Frame: Day 1 - Day 29 ]
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
|Contact: Reference Study ID Number: CP40559 www.roche.com/about_roche/roche_worldwide.htm||888-662-6728 (U.S. and Canada)||firstname.lastname@example.org|