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

540 Participants Needed

Trial Summary

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.

How is the drug Xofluza different from other flu treatments?

Xofluza (baloxavir marboxil) is unique because it is a single-dose oral medication that works by inhibiting a specific protein in the flu virus, stopping it from replicating. This is different from other flu treatments like oseltamivir, which require multiple doses and work by a different mechanism. ^{1 2 3 4 5}

What is the purpose of this trial?

The goal of this prospective, interventional, single-center study is to assess whether the early detection of Influenza with smart wearable device algorithms and alerting, rapid testing, and subsequent Baloxavir treatment demonstrate better post-infection outcomes versus publicly available- and Centers for Disease Control (CDC)-derived national statistics for equivalent household populations as well as pediatric kidney, heart, liver, lung transplant recipients and waitlisted patients.

Research Team

Matthew O Connor, MD

Principal Investigator

Children's Hospital of Philadelphia

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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]

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]

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]

References

1. Netherlands pubmed.ncbi.nlm.nih.gov

Cost-effectiveness of baloxavir marboxil compared to laninamivir for the treatment of influenza in Japan. [2021]

2. New Zealand pubmed.ncbi.nlm.nih.gov

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

3. England pubmed.ncbi.nlm.nih.gov

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

4. United States 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. [2021]

5. Switzerland pubmed.ncbi.nlm.nih.gov

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