Xofluza for Flu

Overseen By Brendan Keating, PhD

Age: Any Age

Sex: Any

Trial Phase: Phase 4

Sponsor: Children's Hospital of Philadelphia

Must be taking: Baloxavir

Must not be taking: Peramivir, Laninamivir, others

Disqualifiers: Pregnancy, Under 5 years, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether using smartwatches for early flu detection and prompt treatment with Baloxavir (also known as Xofluza, an antiviral medication) improves recovery compared to national averages. It targets individuals who have had organ transplants and their household members. Participants should be transplant recipients or on a transplant waitlist, aged 5 or older, and willing to wear a smartwatch. The study focuses on how early detection and treatment enhance outcomes for these groups. As a Phase 4 trial, this research aims to understand how the already FDA-approved and effective treatment benefits more patients, offering participants a chance to contribute to valuable insights.

Will I have to stop taking my current medications?

The trial requires participants to stop taking certain influenza antiviral drugs, except for oseltamivir and baloxavir. If you are taking other influenza antivirals like peramivir, laninamivir, zanamivir, rimantadine, umifenovir, or amantadine, you will need to stop them to participate.

What is the safety track record for Baloxavir Marboxil?

Research has shown that Baloxavir Marboxil, a flu treatment, is generally safe for individuals aged 12 and older. The FDA has approved it for this age group. In studies, most participants tolerated the treatment well, experiencing only mild side effects such as headaches and nausea. However, limited information exists about its use in younger children aged 5 to under 12, particularly those at high risk for flu complications. It is important to note that Baloxavir Marboxil does not prevent bacterial infections that can accompany the flu. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Baloxavir marboxil is unique because it targets the flu virus differently than most other treatments, like oseltamivir (Tamiflu). While many flu medications work by inhibiting the virus's ability to spread, baloxavir marboxil stops the virus from replicating by blocking an enzyme essential for its reproduction. This means it can potentially reduce flu symptoms faster and may require only a single dose. Researchers are excited because these features could lead to more efficient and convenient flu management, especially for vulnerable groups like transplant recipients and their families.

What is the effectiveness track record for Baloxavir Marboxil in treating the flu?

Research has shown that Baloxavir Marboxil, also known as Xofluza, effectively treats the flu. In this trial, participants in certain groups will receive a single dose of Baloxavir. One study found that a single dose of Baloxavir reduced the chance of spreading the flu to others more than a placebo did. Another study showed that in teenagers, Xofluza helped flu symptoms resolve 42% faster, or about 1.6 days sooner, compared to those who didn't take it. Additionally, Xofluza has been proven safe and effective for children as young as one year old. These findings support its ability to manage flu symptoms and limit its spread. ^{6 7 8 9 10}

Who Is on the Research Team?

Matthew O Connor, MD

Principal Investigator

Children's Hospital of Philadelphia

Are You a Good Fit for This Trial?

This trial is for individuals aged 5 or older, including those who have had a transplant (kidney, heart, liver, lung) and their non-transplanted household members. Participants must be willing to wear a smartwatch and take an at-home test for Influenza A or B. Those with allergies to Baloxavir, under 40 kg in weight, pregnant or breastfeeding women, and anyone on mechanical ventilation are excluded.

Inclusion Criteria

My doctor has prescribed Baloxavir for flu prevention after exposure.

I started taking Baloxavir within 48 hours after my symptoms began.

I am a transplant recipient of a kidney, heart, liver, or lung and am 5 years old or older.

Exclusion Criteria

Population 1: If the subject is unable or unwilling to consent

Population 1: If the subject requires mechanical ventilation at time of enrollment

I weigh less than 40 kg.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Monitoring

Participants wear smart wearable devices to monitor biometrics including heart rate and body temperature for early infection detection

Ongoing until infection detection

Continuous monitoring

Treatment

Participants receive a single dose of Baloxavir if positive for Influenza A/B

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including hospital stay length, oxygen requirement, and respiratory failure

30 days

Daily symptom forms

What Are the Treatments Tested in This Trial?

Interventions

• Baloxavir Marboxil

Trial Overview

The study tests if early detection of Influenza using smartwatches followed by rapid testing and treatment with Baloxavir can improve outcomes compared to national statistics. It includes pediatric transplant recipients/waitlisted patients at CHOP.

How Is the Trial Designed?

Group I: Population 3: Household Members (Non-Transplant) Experimental Treatment 1 Intervention

Non-Transplanted Household Members aged 5 years or older will receive a single dose of Baloxavir marboxil.

Group II: Population 2: Waitlisted Patients for Transplant Experimental Treatment 1 Intervention

Participants who are waitlisted CHOP kidney, heart, liver or lung single or multiple transplant recipients aged 5 years or older will receive a single dose of Baloxavir marboxil.

Group III: Population 1: Transplant Recipients Experimental Treatment 1 Intervention

Participants who are a CHOP kidney, heart, liver or lung single or multiple transplant recipients aged 5 years or older will receive a single dose of Baloxavir.

Group IV: Population 4: Non-Baloxavir treatment subjects Active Control 1 Intervention

CHOP transplant recipients and all other non-transplanted household members who are 2-4 years of age. Subjects 5 years and up who are Influenza positive but whom do not receive Baloxavir treatment

Baloxavir Marboxil is already approved in United States, Japan, European Union for the following indications:

Approved in United States as Xofluza for:

• Acute uncomplicated influenza within 2 days of illness onset in people aged ≥5 years who are otherwise healthy, or in people aged ≥12 years who are at high risk of developing influenza-related complications

Approved in Japan as Xofluza for:

• Influenza A and B virus infections

Approved in European Union as Xofluza for:

• Treatment of uncomplicated influenza in adults and adolescents aged 12 years and older weighing at least 40 kg

Who Is Running the Clinical Trial?

Children's Hospital of Philadelphia

Lead Sponsor

Trials

749

Recruited

11,400,000+

Genentech, Inc.

Industry Sponsor

Trials

1,578

Recruited

569,000+

Ashley Magargee

Genentech, Inc.

Chief Executive Officer since 2024

MBA from Harvard University, BA from Princeton University

Levi Garraway

Genentech, Inc.

Chief Medical Officer since 2021

MD, PhD

Published Research Related to This Trial

Baloxavir marboxil is a cost-effective antiviral treatment for influenza in otherwise healthy adults in Japan, showing a small gain in quality-adjusted life-years (QALYs) compared to laninamivir, with an incremental cost per QALY gained of ¥2,231,260, which is below the willingness-to-pay threshold of ¥5,000,000/QALY.

The study indicates that the additional costs of baloxavir are offset by avoided drug-related adverse events (DRAEs), and the probability of baloxavir being cost-effective is estimated at 64%, suggesting it may be a favorable option for influenza treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cost-effectiveness of baloxavir marboxil compared to laninamivir for the treatment of influenza in Japan. Skrzeczek, A., Ikeoka, H., Hirotsu, N., et al.[2021]

In a study involving 115,238 children aged 7-15 years, baloxavir marboxil did not lead to increased medical resource use compared to neuraminidase inhibitors (NAIs) within 9 days of treatment, indicating its safety in terms of healthcare-seeking behavior.

However, baloxavir was associated with higher medical resource use compared to oseltamivir, suggesting that while it is effective, it may lead to more follow-up care than this specific NAI.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Association of baloxavir marboxil prescription with subsequent medical resource utilization among school-aged children with influenza. Takeuchi, M., Kawakami, K.[2021]

Baloxavir marboxil is an effective single-dose oral treatment for acute uncomplicated influenza in patients aged 12 and older, showing similar efficacy to oseltamivir and faster reduction of viral load.

It works by inhibiting the influenza virus's polymerase acidic protein, and while it is well tolerated, monitoring for resistant virus strains is important due to emerging variants.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Baloxavir Marboxil: A Review in Acute Uncomplicated Influenza. Shirley, M.[2021]

Baloxavir marboxil is a well-tolerated new treatment for seasonal flu, effective against both influenza A and B viruses, although B strains show some resistance due to genetic variations.

The drug is currently in the post-marketing phase, and its safety profile is being monitored, with potential future applications being explored in clinical trials for COVID-19 alongside favipiravir.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Baloxavir Marboxil: An Original New Drug against Influenza. Dufrasne, F.[2022]

In a study of 339,007 patients, baloxavir marboxil was associated with a lower incidence of hospitalization compared to oseltamivir and zanamivir, suggesting it may be a more effective treatment for influenza.

Patients treated with baloxavir required fewer additional antiinfluenza drugs and had a lower likelihood of needing antibacterial treatments, indicating its potential for reducing complications associated with influenza.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of Hospitalization Incidence in Influenza Outpatients Treated With Baloxavir Marboxil or Neuraminidase Inhibitors: A Health Insurance Claims Database Study. Komeda, T., Takazono, T., Hosogaya, N., et al.[2021]

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40267424/

Efficacy of Baloxavir Treatment in Preventing Transmission ...

Treatment with a single oral dose of baloxavir led to a lower incidence of transmission of influenza virus to close contacts than placebo.

2.

roche.com

roche.com/media/releases/med-cor-2025-04-25

New England Journal of Medicine publishes phase III data ...

The trial met its primary endpoint, showing a single, oral dose of Xofluza taken by people infected with influenza reduced the odds of untreated ...

3.

xofluza-hcp.com

xofluza-hcp.com/efficacy/otherwise-healthy.html

XOFLUZA® (baloxavir marboxil) Otherwise Healthy Patients

42% (1.6 days) faster relief from flu symptoms (in patients aged 12 to 17 years) with single-dose XOFLUZA compared with placebo.1. For subjects aged 12 to 17 ...

4.

gene.com

gene.com/media/press-releases/15037/2024-09-18/positive-phase-iii-results-show-xofluza-

Genentech: Press Releases | Wednesday, Sep 18, 2024

Data from the CENTERSTONE study shows single-dose Xofluza reduces transmission of influenza from an infected person to household members.

5.

journals.lww.com

journals.lww.com/pidj/fulltext/2025/07000/a_phase_3_safety_and_efficacy_study_of_baloxavir.7.aspx

A Phase 3 Safety and Efficacy Study of Baloxavir Marboxil...

Baloxavir marboxil (baloxavir) inhibits influenza virus cap-dependent endonuclease and has demonstrated safety and efficacy in children 1–<12 years of age.

6.

xofluza-hcp.com

xofluza-hcp.com/safety.html

Safety profile for flu treatment and post-exposure prophylaxis

There are limited safety data in patients 5 to <12 years at high risk of developing influenza complications.

7.

xofluza.com

xofluza.com/safety.html

Things you need to know about XOFLUZA safety

XOFLUZA does not prevent bacterial infections that may happen with the flu. It is not known if XOFLUZA is safe and effective for the treatment and prevention ...

8.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2018/210854s000lbl.pdf

Baloxavir marboxil - accessdata.fda.gov

The safety and effectiveness of XOFLUZA for the treatment of influenza have been established in pediatric patients 12 years of age and older weighing at least ...

9.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12107739/

Safety evaluation of baloxavir marboxil

As a novel anti-influenza agent, baloxavir marboxil lacks real-world safety data in large populations. Therefore, this study aimed to ...

10.

ema.europa.eu

ema.europa.eu/en/documents/product-information/xofluza-epar-product-information_en.pdf

Xofluza, INN-baloxavir marboxil - EMA

The predominant influenza virus strain in this study was the A/H3 subtype. The primary objective was to compare the safety of a single dose of baloxavir.