InfluenzaPhase 2/3NCT05170009

Baloxavir in Combination With Oseltamivir in Allogenic Bone Marrow Transplant Recipients With Influenza

A Phase 2/3 study testing Baloxavir Marboxil and Oseltamivir in people with Influenza, run by Weill Medical College of Cornell University at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Influenza
What is being tested?: Baloxavir Marboxil and Oseltamivir
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 2 participants
Where: 1 siteUnited States
Study dates: 22 Apr 2022to 7 Feb 2024
Phase: Phase 2/3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Adult patients: Signed informed consent by any patient capable of giving consent, or, where the patient is not capable of giving consent, by his or her legal/authorized representative
Age greater than or equal to 18 years at the time of signing the Informed Consent Form/Assent Form
Ability to comply with the study protocol, in the investigator's judgment
Have received allogeneic bone marrow transplant
Tested positive for influenza infection after the onset of symptoms using a polymerase chain reaction (PCR)-based diagnostic assay.
Presence of (a) fever (≥38.0 °C per tympanic or rectal thermometer; ≥ 37.5 °C per axillary, oral or forehead/temporal thermometer) or (b) any influenza symptoms (cough, sore throat, nasal congestion, headache, feverishness or chills, muscle or joint pain, fatigue).

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The time interval between the diagnosis of influenza and the pre-dose examinations is 48 hours or less.
For women of childbearing potential: Agreement to remain abstinent (refrain from heterosexual intercourse):
Women must remain abstinent or use contraceptive methods with a failure rate of < 1% per year during the treatment period and for 28 days after the last dose of study treatment. Hormonal contraceptive methods must be supplemented by a barrier method. A woman is considered to be of childbearing potential if she is postmenarcheal, has not reached a postmenopausal state greater than or equal to 12 continuous months of amenorrhea with no identified cause other than menopause), and has not undergone surgical sterilization (removal of ovaries and/or uterus). Examples of contraceptive methods with a failure rate of < 1% per year include bilateral tubal ligation, male sterilization, hormonal contraceptives that inhibit ovulation, hormone-releasing intrauterine devices, and copper intrauterine devices. The reliability of sexual abstinence should be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or postovulation methods) and withdrawal are not acceptable methods of contraception.

Who cannot join

Patients who have received more than 48 hours of antiviral treatment for the current influenza infection prior to screening
Patients who have received Baloxavir for the current influenza infection
Known contraindication to neuraminidase inhibitors
Patients weighing < 40 kg
Patients unable to swallow tablets
Patients with known severe renal impairment (estimated glomerular filtration rate < 30 mL/min/1.73 m2) or receiving continuous renal replacement therapy, hemodialysis, peritoneal dialysis

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Patients with any of the following laboratory abnormalities detected within 24 hours prior to or during screening (according to local laboratory reference ranges: ALT or AST level > 5 times the upper limit of normal (ULN) OR ALT or AST > 3 times the ULN and total bilirubin level > 2 times the ULN
Pregnant or breastfeeding, or positive pregnancy test in a predose examination, or intending to become pregnant during the study or within 28 days after the last dose of study treatment
Exposure to an investigational drug within 5 half-lives or 30 days (whichever is longer) of randomization
Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study
Known hypersensitivity to baloxavir marboxil or the drug product excipients
Known COVID-19 coinfection
Unwilling to undergo nasopharyngeal (NP) swabs as per study schedule

Trial locations and contacts

1 site in 1 country.

United States1 site

Weill Cornell Medicine
New York, New York, 10065

Other trials in United States

About this study

Official title: Randomized Double-blind, Placebo-controlled Single Center Pilot Study to Evaluate the Efficacy and Safety of Baloxavir in Combination With Oseltamivir in Adult Allogeneic Bone Marrow Transplant Recipients With Influenza

This is a randomized, double-blind, placebo-controlled pilot study of the efficacy and safety of baloxavir in combination with oseltamivir (standard of care) for the treatment of influenza in allogeneic stem cell transplant patients. Although there are no data about this treatment option currently available, the investigator hypothesizes that combination therapy may be more effective in clearing influenza virus infection and decreasing the rate of emergence of resistant influenza in immunocompromised human hosts.

Read the detailed description

This is a randomized double-blind placebo-controlled pilot study of the efficacy and safety of baloxavir in combination with oseltamivir (standard of care) for the treatment of influenza in allogeneic stem cell transplant patients. 30 SCT recipients will take part in the study. Participants with be randomly assigned (1:1) to either baloxavir + oseltamivir or baloxavir-matched placebo +oseltamivir. Before randomization, patients will be stratified by hospitalization status and influenza type A (yes/no).

Patients in the baloxavir combination arm will receive weight-adjusted baloxavir (40 mg for patients weighing <80 kg and 80 mg for those weighing ≥80 kg) at baseline and at day 4 and day 7. They will also receive oseltamivir 75 mg twice daily for 10 days. Patients in the baloxavir-matched placebo + oseltamivir arm will receive baloxavir-matched placebo at baseline and at day 3 and day 7and oseltamivir 75 mg twice daily for 10 days.

Conditions: Influenza
Being tested: Baloxavir Marboxil, Placebo, Oseltamivir
Keywords: Influenza, Bone marrow transplant, Flu, Baloxavir Marboxil, Xofluza, Oseltamivir
Medical subject headings: Influenza, Human

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Baloxavir MarboxilDrug
Also called Xofluza
Weight-adjusted Baloxavir Marboxil (40 mg for patients weighing <80 kg and 80 mg for those weighing ≥80 kg) at baseline, day 4, and day 7.
PlaceboDrug
Placebo at baseline, day 4, and day 7.
OseltamivirDrug
Also called Tamiflu
Oseltamivir 75 mg twice daily for 10 days.

Study groups in NCT05170009
Study group	Role	What they receive
Active and standard of care	Active comparator	Active Baloxavir Marboxil and standard of care Oseltamivir Receives: Drug: Baloxavir Marboxil; Drug: Oseltamivir
Placebo and standard of care	Placebo comparator	Placebo-matched Baloxavir Marboxil and standard of care Oseltamivir Receives: Drug: Placebo; Drug: Oseltamivir

What is being measured

The primary outcome is the main question the study is designed to answer.

Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Quantitative Real Time Polymerase Chain Reaction.
Influenza viral RNA loads will be measured by quantitative real time polymerase chain reaction.
Measured: Baseline; Day 10
Between-treatment-arm Difference in the Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Quantitative Real Time Polymerase Chain Reaction.
Influenza viral load will be measured by the quantitative real time polymerase chain reaction.
Measured: Baseline; Day 10
Between-treatment-arm Difference in the Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Influenza Plaque Assay (Replicating Virus).
Influenza viral load will be measured by the influenza plaque assay.
Measured: Baseline; Day 10

Secondary outcomes (11)

Change of Influenza Viral RNA Load From Baseline at Day 4 and Day 7 and 10 in Each Treatment Arm
Influenza viral RNA load will be measured by quantitative real time polymerase chain reaction.
Measured: Baseline; Day 4; Day 7; Day 10
Difference in Change of Influenza Viral Loads From Baseline at Day 4, 7 and 10 Between the Two Treatment Arms as Measured by Quantitative Real Time Polymerase Chain Reaction
Influenza viral RNA load will be measured by quantitative real time polymerase chain reaction.
Measured: Baseline; Day 4; Day 7; Day 10
Change of Influenza Viral Loads From Baseline at Day 4, 7 and 10 as Measured by Influenza Plaque Assay (Replicating Virus) in Each Treatment Arm
Influenza viral load will be measured by the influenza plaque assay (replicating virus).
Measured: Baseline; Day 4; Day 7; Day 10
Difference in Change of Influenza Viral Loads From Baseline at Day 4, 7 and 10 Between the Two Treatment Arms as Measured by Influenza Plaque Assay (Replicating Virus)
Influenza viral load will be measured by the influenza plaque assay (replicating virus).
Measured: Baseline; Day 4; Day 7; Day 10
Time to Improvement of Individual Influenza Symptoms as Assessed by Patient-reported Outcome Measures on a Single Scale
Patients will self-assess the severity of 7 influenza-associated symptoms on a 4-point single scale with 0 indicating no symptoms and higher scores indicating mild, moderate, and severe symptoms. Time to improvement of individual influenza symptoms are defined as the time from the start of treatment to the time when each of the influenza symptoms are alleviated, maintained, or improved for a duration of at least 21.5 hours. These are defined as: pre-existing symptoms (cough, fatigue, or muscle/join pain that existed prior to influenza) that were worse at baseline and had improved at least 1 point from baseline; pre-existing symptoms not worse at baseline that maintained baseline severity; and new symptoms that were alleviated, defined as a symptom score of non (0) or mild (1).
Measured: Baseline to Day 30
Percentage of Patients Who Experience Each Influenza-related Complications: Hospitalization, Death, Sinusitis, Otitis Media, Bronchitis, and Radiologically-confirmed Pneumonia as an Adverse Event After the Initiation of Study Treatment
A composite score of multiple measures will be used and calculated by count of patients who experience each influenza-related complications. Adverse events will only include those that are determined to be related to the study drug.
Measured: Day 1 to Day 30
Time to Return to Preinfluenza Health Status
Preinfluenza health status will be measured on a score from 0 (worst possible health) to 10 (normal health [for someone your age and condition]).
Measured: Day 1 to Day 30
Time to Viral Clearance, as Assessed by Difference in Percentage of Participants Positive by Influenza Plaque Assay at Each Time-point (in Each Treatment Group)
Time to viral clearance will be assessed by percentage of participants positive by influenza plaque assay at each time-point.
Measured: Baseline to Day 30
Time to Viral Clearance, as Assessed by Difference in Percentage of Participants Positive by Quantitative Real Time Polymerase Chain Reaction
Time to viral clearance will be assessed by percentage of participants positive by qPCR at each time-point.
Measured: Baseline to Day 30
Change of Treatment-emergent Variants of Neuraminidase and Polymerase Known to Confer Antiviral Resistance to Oseltamivir in Each Arm by Direct Next-generation Sequencing Symptoms
Treatment-emergent variants will be identified using direct next-generation sequencing of a comprehensive panel of genes.
Measured: Baseline to Day 30
Percentage of Participants With Adverse Events (AEs)
Adverse events will only include those that are determined to be related to the study drug and will assess the safety and tolerability of Baloxavir in Combination with SOC treatment.
Measured: Day 1 to Day 15

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

22 Apr 20227 Feb 2024

First posted27 Dec 2021
Study start22 Apr 2022
Primary completion17 Jan 2024
Study completion7 Feb 2024

Why it stopped: Slow enrollment rate

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (18)Papers the sponsor has linked to this study.
Other trials for InfluenzaStudies of the same condition, open and under way.
More trials run by Weill Medical College of Cornell UniversityThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2/3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 2 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05170009
Sponsor's protocol ID: 20-12023076
Status last verified: Feb 2025
First submitted: 9 Dec 2021
Last updated: 26 Feb 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Burnham P, Khush K, De Vlaminck I. Myriad Applications of Circulating Cell-Free DNA in Precision Organ Transplant Monitoring. Ann Am Thorac Soc. 2017 Sep;14(Supplement_3):S237-S241. doi: 10.1513/AnnalsATS.201608-634MG.
PubMed 28945480
de Jong MD, Ison MG, Monto AS, Metev H, Clark C, O'Neil B, Elder J, McCullough A, Collis P, Sheridan WP. Evaluation of intravenous peramivir for treatment of influenza in hospitalized patients. Clin Infect Dis. 2014 Dec 15;59(12):e172-85. doi: 10.1093/cid/ciu632. Epub 2014 Aug 12.
PubMed 25115871
De Vlaminck I, Martin L, Kertesz M, Patel K, Kowarsky M, Strehl C, Cohen G, Luikart H, Neff NF, Okamoto J, Nicolls MR, Cornfield D, Weill D, Valantine H, Khush KK, Quake SR. Noninvasive monitoring of infection and rejection after lung transplantation. Proc Natl Acad Sci U S A. 2015 Oct 27;112(43):13336-41. doi: 10.1073/pnas.1517494112. Epub 2015 Oct 12.
PubMed 26460048
De Vlaminck I, Valantine HA, Snyder TM, Strehl C, Cohen G, Luikart H, Neff NF, Okamoto J, Bernstein D, Weisshaar D, Quake SR, Khush KK. Circulating cell-free DNA enables noninvasive diagnosis of heart transplant rejection. Sci Transl Med. 2014 Jun 18;6(241):241ra77. doi: 10.1126/scitranslmed.3007803.
PubMed 24944192
Fiore AE, Fry A, Shay D, Gubareva L, Bresee JS, Uyeki TM; Centers for Disease Control and Prevention (CDC). Antiviral agents for the treatment and chemoprophylaxis of influenza --- recommendations of the Advisory Committee on Immunization Practices (ACIP). MMWR Recomm Rep. 2011 Jan 21;60(1):1-24.
PubMed 21248682
Fukao K, Ando Y, Noshi T, Kitano M, Noda T, Kawai M, Yoshida R, Sato A, Shishido T, Naito A. Baloxavir marboxil, a novel cap-dependent endonuclease inhibitor potently suppresses influenza virus replication and represents therapeutic effects in both immunocompetent and immunocompromised mouse models. PLoS One. 2019 May 20;14(5):e0217307. doi: 10.1371/journal.pone.0217307. eCollection 2019.
PubMed 31107922
Fukao K, Noshi T, Yamamoto A, Kitano M, Ando Y, Noda T, Baba K, Matsumoto K, Higuchi N, Ikeda M, Shishido T, Naito A. Combination treatment with the cap-dependent endonuclease inhibitor baloxavir marboxil and a neuraminidase inhibitor in a mouse model of influenza A virus infection. J Antimicrob Chemother. 2019 Mar 1;74(3):654-662. doi: 10.1093/jac/dky462.
PubMed 30476172
Hayden FG, Sugaya N, Hirotsu N, Lee N, de Jong MD, Hurt AC, Ishida T, Sekino H, Yamada K, Portsmouth S, Kawaguchi K, Shishido T, Arai M, Tsuchiya K, Uehara T, Watanabe A; Baloxavir Marboxil Investigators Group. Baloxavir Marboxil for Uncomplicated Influenza in Adults and Adolescents. N Engl J Med. 2018 Sep 6;379(10):913-923. doi: 10.1056/NEJMoa1716197.
PubMed 30184455
Harper SA, Bradley JS, Englund JA, File TM, Gravenstein S, Hayden FG, McGeer AJ, Neuzil KM, Pavia AT, Tapper ML, Uyeki TM, Zimmerman RK; Expert Panel of the Infectious Diseases Society of America. Seasonal influenza in adults and children--diagnosis, treatment, chemoprophylaxis, and institutional outbreak management: clinical practice guidelines of the Infectious Diseases Society of America. Clin Infect Dis. 2009 Apr 15;48(8):1003-32. doi: 10.1086/598513.
PubMed 19281331
Hu Y, Lu S, Song Z, Wang W, Hao P, Li J, Zhang X, Yen HL, Shi B, Li T, Guan W, Xu L, Liu Y, Wang S, Zhang X, Tian D, Zhu Z, He J, Huang K, Chen H, Zheng L, Li X, Ping J, Kang B, Xi X, Zha L, Li Y, Zhang Z, Peiris M, Yuan Z. Association between adverse clinical outcome in human disease caused by novel influenza A H7N9 virus and sustained viral shedding and emergence of antiviral resistance. Lancet. 2013 Jun 29;381(9885):2273-9. doi: 10.1016/S0140-6736(13)61125-3. Epub 2013 May 29.
PubMed 23726392
Lee N, Chan PK, Hui DS, Rainer TH, Wong E, Choi KW, Lui GC, Wong BC, Wong RY, Lam WY, Chu IM, Lai RW, Cockram CS, Sung JJ. Viral loads and duration of viral shedding in adult patients hospitalized with influenza. J Infect Dis. 2009 Aug 15;200(4):492-500. doi: 10.1086/600383.
PubMed 19591575
Lee N, Ison MG. Diagnosis, management and outcomes of adults hospitalized with influenza. Antivir Ther. 2012;17(1 Pt B):143-57. doi: 10.3851/IMP2059. Epub 2012 Feb 3.
PubMed 22311561
Marty FM, Vidal-Puigserver J, Clark C, Gupta SK, Merino E, Garot D, Chapman MJ, Jacobs F, Rodriguez-Noriega E, Husa P, Shortino D, Watson HA, Yates PJ, Peppercorn AF. Intravenous zanamivir or oral oseltamivir for hospitalised patients with influenza: an international, randomised, double-blind, double-dummy, phase 3 trial. Lancet Respir Med. 2017 Feb;5(2):135-146. doi: 10.1016/S2213-2600(16)30435-0. Epub 2017 Jan 14.
PubMed 28094141
Muthuri SG, Venkatesan S, Myles PR, Leonardi-Bee J, Al Khuwaitir TS, Al Mamun A, Anovadiya AP, Azziz-Baumgartner E, Baez C, Bassetti M, Beovic B, Bertisch B, Bonmarin I, Booy R, Borja-Aburto VH, Burgmann H, Cao B, Carratala J, Denholm JT, Dominguez SR, Duarte PA, Dubnov-Raz G, Echavarria M, Fanella S, Gao Z, Gerardin P, Giannella M, Gubbels S, Herberg J, Iglesias AL, Hoger PH, Hu X, Islam QT, Jimenez MF, Kandeel A, Keijzers G, Khalili H, Knight M, Kudo K, Kusznierz G, Kuzman I, Kwan AM, Amine IL, Langenegger E, Lankarani KB, Leo YS, Linko R, Liu P, Madanat F, Mayo-Montero E, McGeer A, Memish Z, Metan G, Mickiene A, Mikic D, Mohn KG, Moradi A, Nymadawa P, Oliva ME, Ozkan M, Parekh D, Paul M, Polack FP, Rath BA, Rodriguez AH, Sarrouf EB, Seale AC, Sertogullarindan B, Siqueira MM, Skret-Magierlo J, Stephan F, Talarek E, Tang JW, To KK, Torres A, Torun SH, Tran D, Uyeki TM, Van Zwol A, Vaudry W, Vidmar T, Yokota RT, Zarogoulidis P; PRIDE Consortium Investigators; Nguyen-Van-Tam JS. Effectiveness of neuraminidase inhibitors in reducing mortality in patients admitted to hospital with influenza A H1N1pdm09 virus infection: a meta-analysis of individual participant data. Lancet Respir Med. 2014 May;2(5):395-404. doi: 10.1016/S2213-2600(14)70041-4. Epub 2014 Mar 19.
PubMed 24815805
Paules C, Subbarao K. Influenza. Lancet. 2017 Aug 12;390(10095):697-708. doi: 10.1016/S0140-6736(17)30129-0. Epub 2017 Mar 13.
PubMed 28302313

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 26 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Randomized Double-blind, Placebo-controlled Single Center Pilot Study to Evaluate the Efficacy and Safety of Baloxavir in Combination With Oseltamivir in Adult Allogeneic Bone Marrow Transplant Recipients With Influenza
ClinicalTrials.gov ID: NCT05170009
Acronym / sponsor ID: 20-12023076
Conditions: Influenza
Keywords: Influenza, Bone marrow transplant, Flu, Baloxavir Marboxil, Xofluza, Oseltamivir
Phase: Phase 2/3
Study type: Interventional
Recruitment status: TerminatedReason stopped: Slow enrollment rate
Enrolment: 2 participants (actual)
Lead sponsor: Weill Medical College of Cornell UniversityUniversity, hospital or other
Collaborators: Genentech, Inc.
Study start: 22 Apr 2022 (actual)
Primary completion: 17 Jan 2024 (actual)
Study completion: 7 Feb 2024 (actual)
First posted: 27 Dec 2021 (actual)
Results: Posted 26 Feb 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Active and standard of care	Active comparator	Drug: Baloxavir Marboxil, Drug: OseltamivirActive Baloxavir Marboxil and standard of care Oseltamivir
Placebo and standard of care	Placebo comparator	Drug: Placebo, Drug: OseltamivirPlacebo-matched Baloxavir Marboxil and standard of care Oseltamivir

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (9 listed)

Adult patients: Signed informed consent by any patient capable of giving consent, or, where the patient is not capable of giving consent, by his or her legal/authorized representative
Age greater than or equal to 18 years at the time of signing the Informed Consent Form/Assent Form
Ability to comply with the study protocol, in the investigator's judgment
Have received allogeneic bone marrow transplant
Tested positive for influenza infection after the onset of symptoms using a polymerase chain reaction (PCR)-based diagnostic assay.

Key exclusion criteria (13 listed)

Patients who have received more than 48 hours of antiviral treatment for the current influenza infection prior to screening
Patients who have received Baloxavir for the current influenza infection
Known contraindication to neuraminidase inhibitors
Patients weighing < 40 kg
Patients unable to swallow tablets

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Baloxavir MarboxilAlso: XofluzaWeight-adjusted Baloxavir Marboxil (40 mg for patients weighing <80 kg and 80 mg for those weighing ≥80 kg) at baseline, day 4, and day 7.	Drug	Active and standard of care
PlaceboPlacebo at baseline, day 4, and day 7.	Drug	Placebo and standard of care
OseltamivirAlso: TamifluOseltamivir 75 mg twice daily for 10 days.	Drug	Active and standard of care, Placebo and standard of care

Primary outcome

Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Quantitative Real Time Polymerase Chain Reaction.
Time frame: Baseline; Day 10
Influenza viral RNA loads will be measured by quantitative real time polymerase chain reaction.
Between-treatment-arm Difference in the Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Quantitative Real Time Polymerase Chain Reaction.
Time frame: Baseline; Day 10
Influenza viral load will be measured by the quantitative real time polymerase chain reaction.
Between-treatment-arm Difference in the Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Influenza Plaque Assay (Replicating Virus).
Time frame: Baseline; Day 10
Influenza viral load will be measured by the influenza plaque assay.

11 secondary outcomes

Change of Influenza Viral RNA Load From Baseline at Day 4 and Day 7 and 10 in Each Treatment Arm
Time frame: Baseline; Day 4; Day 7; Day 10
Difference in Change of Influenza Viral Loads From Baseline at Day 4, 7 and 10 Between the Two Treatment Arms as Measured by Quantitative Real Time Polymerase Chain Reaction
Time frame: Baseline; Day 4; Day 7; Day 10
Change of Influenza Viral Loads From Baseline at Day 4, 7 and 10 as Measured by Influenza Plaque Assay (Replicating Virus) in Each Treatment Arm
Time frame: Baseline; Day 4; Day 7; Day 10
Difference in Change of Influenza Viral Loads From Baseline at Day 4, 7 and 10 Between the Two Treatment Arms as Measured by Influenza Plaque Assay (Replicating Virus)
Time frame: Baseline; Day 4; Day 7; Day 10
Time to Improvement of Individual Influenza Symptoms as Assessed by Patient-reported Outcome Measures on a Single Scale
Time frame: Baseline to Day 30
Percentage of Patients Who Experience Each Influenza-related Complications: Hospitalization, Death, Sinusitis, Otitis Media, Bronchitis, and Radiologically-confirmed Pneumonia as an Adverse Event After the Initiation of Study Treatment
Time frame: Day 1 to Day 30
Time to Return to Preinfluenza Health Status
Time frame: Day 1 to Day 30
Time to Viral Clearance, as Assessed by Difference in Percentage of Participants Positive by Influenza Plaque Assay at Each Time-point (in Each Treatment Group)
Time frame: Baseline to Day 30
Time to Viral Clearance, as Assessed by Difference in Percentage of Participants Positive by Quantitative Real Time Polymerase Chain Reaction
Time frame: Baseline to Day 30
Change of Treatment-emergent Variants of Neuraminidase and Polymerase Known to Confer Antiviral Resistance to Oseltamivir in Each Arm by Direct Next-generation Sequencing Symptoms
Time frame: Baseline to Day 30
Percentage of Participants With Adverse Events (AEs)
Time frame: Day 1 to Day 15

Results and safety

Results posted to the registry on 26 Feb 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Active and Standard of Care	0	0
Placebo and Standard of Care	2	1

Primary outcome: Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Quantitative Real Time Polymerase Chain Reaction.

Time frame: Baseline; Day 10. Number. Unit: copies per mL

Measure	Active and Standard of Care n = 0	Placebo and Standard of Care n = 1
Baseline	–	2300000
Day 10	–	0

Population analysed: The only patient that completed the study, was randomized into the placebo arm.

Primary outcome: Between-treatment-arm Difference in the Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Quantitative Real Time Polymerase Chain Reaction.

Time frame: Baseline; Day 10. Number. Unit: copies per mL

Measure	Active and Standard of Care n = 0	Placebo and Standard of Care n = 1
Baseline	–	2300000
Day 10	–	0

Population analysed: The only patient that completed the study, was randomized into the placebo arm. Difference cannot be calculated as one arm has zero patients.

Primary outcome: Between-treatment-arm Difference in the Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Influenza Plaque Assay (Replicating Virus).

Time frame: Baseline; Day 10. Number. Unit: PFU per mL

Measure	Active and Standard of Care n = 0	Placebo and Standard of Care n = 1
Baseline	–	2300000
Day 10	–	0

Population analysed: The only patient that completed the study, was randomized into the placebo arm. Difference cannot be calculated as one arm has zero patients.

11 secondary outcomes are reported in the full results.

Adverse events

Time frame: 1 month. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Active and Standard of Care	0 of 0	0 of 0	0
Placebo and Standard of Care	1 of 2 (50%)	1 of 2 (50%)	1 of 2

Limitations and caveats (from the sponsor): Due to change in patterns of respiratory viruses, and low or no flu circulating, we were unable to enroll the target number of participants. The only participants enrolled were the 2 in the placebo arm, 1 of whom withdrew early on. No comparison is possible with only 1 placebo participant.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 28945480Burnham P, Khush K, De Vlaminck I. Myriad Applications of Circulating Cell-Free DNA in Precision Organ Transplant Monitoring. Ann Am Thorac Soc. 2017 Sep;14(Supplement_3):S237-S241. doi: 10.1513/AnnalsATS.201608-634MG.
Background: PubMed 25115871de Jong MD, Ison MG, Monto AS, Metev H, Clark C, O'Neil B, Elder J, McCullough A, Collis P, Sheridan WP. Evaluation of intravenous peramivir for treatment of influenza in hospitalized patients. Clin Infect Dis. 2014 Dec 15;59(12):e172-85. doi: 10.1093/cid/ciu632. Epub 2014 Aug 12.
Background: PubMed 26460048De Vlaminck I, Martin L, Kertesz M, Patel K, Kowarsky M, Strehl C, Cohen G, Luikart H, Neff NF, Okamoto J, Nicolls MR, Cornfield D, Weill D, Valantine H, Khush KK, Quake SR. Noninvasive monitoring of infection and rejection after lung transplantation. Proc Natl Acad Sci U S A. 2015 Oct 27;112(43):13336-41. doi: 10.1073/pnas.1517494112. Epub 2015 Oct 12.
Background: PubMed 24944192De Vlaminck I, Valantine HA, Snyder TM, Strehl C, Cohen G, Luikart H, Neff NF, Okamoto J, Bernstein D, Weisshaar D, Quake SR, Khush KK. Circulating cell-free DNA enables noninvasive diagnosis of heart transplant rejection. Sci Transl Med. 2014 Jun 18;6(241):241ra77. doi: 10.1126/scitranslmed.3007803.
Background: PubMed 21248682Fiore AE, Fry A, Shay D, Gubareva L, Bresee JS, Uyeki TM; Centers for Disease Control and Prevention (CDC). Antiviral agents for the treatment and chemoprophylaxis of influenza --- recommendations of the Advisory Committee on Immunization Practices (ACIP). MMWR Recomm Rep. 2011 Jan 21;60(1):1-24.
Background: PubMed 31107922Fukao K, Ando Y, Noshi T, Kitano M, Noda T, Kawai M, Yoshida R, Sato A, Shishido T, Naito A. Baloxavir marboxil, a novel cap-dependent endonuclease inhibitor potently suppresses influenza virus replication and represents therapeutic effects in both immunocompetent and immunocompromised mouse models. PLoS One. 2019 May 20;14(5):e0217307. doi: 10.1371/journal.pone.0217307. eCollection 2019.
Background: PubMed 30476172Fukao K, Noshi T, Yamamoto A, Kitano M, Ando Y, Noda T, Baba K, Matsumoto K, Higuchi N, Ikeda M, Shishido T, Naito A. Combination treatment with the cap-dependent endonuclease inhibitor baloxavir marboxil and a neuraminidase inhibitor in a mouse model of influenza A virus infection. J Antimicrob Chemother. 2019 Mar 1;74(3):654-662. doi: 10.1093/jac/dky462.
Background: PubMed 30184455Hayden FG, Sugaya N, Hirotsu N, Lee N, de Jong MD, Hurt AC, Ishida T, Sekino H, Yamada K, Portsmouth S, Kawaguchi K, Shishido T, Arai M, Tsuchiya K, Uehara T, Watanabe A; Baloxavir Marboxil Investigators Group. Baloxavir Marboxil for Uncomplicated Influenza in Adults and Adolescents. N Engl J Med. 2018 Sep 6;379(10):913-923. doi: 10.1056/NEJMoa1716197.
Background: PubMed 19281331Harper SA, Bradley JS, Englund JA, File TM, Gravenstein S, Hayden FG, McGeer AJ, Neuzil KM, Pavia AT, Tapper ML, Uyeki TM, Zimmerman RK; Expert Panel of the Infectious Diseases Society of America. Seasonal influenza in adults and children--diagnosis, treatment, chemoprophylaxis, and institutional outbreak management: clinical practice guidelines of the Infectious Diseases Society of America. Clin Infect Dis. 2009 Apr 15;48(8):1003-32. doi: 10.1086/598513.
Background: PubMed 23726392Hu Y, Lu S, Song Z, Wang W, Hao P, Li J, Zhang X, Yen HL, Shi B, Li T, Guan W, Xu L, Liu Y, Wang S, Zhang X, Tian D, Zhu Z, He J, Huang K, Chen H, Zheng L, Li X, Ping J, Kang B, Xi X, Zha L, Li Y, Zhang Z, Peiris M, Yuan Z. Association between adverse clinical outcome in human disease caused by novel influenza A H7N9 virus and sustained viral shedding and emergence of antiviral resistance. Lancet. 2013 Jun 29;381(9885):2273-9. doi: 10.1016/S0140-6736(13)61125-3. Epub 2013 May 29.
Background: PubMed 19591575Lee N, Chan PK, Hui DS, Rainer TH, Wong E, Choi KW, Lui GC, Wong BC, Wong RY, Lam WY, Chu IM, Lai RW, Cockram CS, Sung JJ. Viral loads and duration of viral shedding in adult patients hospitalized with influenza. J Infect Dis. 2009 Aug 15;200(4):492-500. doi: 10.1086/600383.
Background: PubMed 22311561Lee N, Ison MG. Diagnosis, management and outcomes of adults hospitalized with influenza. Antivir Ther. 2012;17(1 Pt B):143-57. doi: 10.3851/IMP2059. Epub 2012 Feb 3.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Mirella Salvatore, MD principal investigator

Weill Medical College of Cornell University

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Baloxavir Marboxil and Oseltamivir in people with Influenza, run by Weill Medical College of Cornell University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 26 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change of Influenza Viral RNA Loads From Baseline at the End of Treatment as Measured by Quantitative Real Time Polymerase Chain Reaction. (time frame: Baseline; Day 10). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05170009, which also lists 18 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Feb 2025.