Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants From Birth to < 1 Year With Influenza-Like Symptoms
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT03653364 |
|
Recruitment Status :
Not yet recruiting
First Posted : August 31, 2018
Last Update Posted : August 31, 2018
|
Sponsor:
Hoffmann-La Roche
Information provided by (Responsible Party):
Hoffmann-La Roche
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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
| 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 :
- 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 :
- 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 ]
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| 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 |