What side effects are associated with this type of intervention?

Baloxavir marboxil, an oral antiviral that inhibits cap-dependent endonuclease activity of the influenza virus, is generally well tolerated. Common side effects include diarrhea and vomiting, with rare reports of hypersensitivity reactions such as anaphylaxis and urticaria. Oseltamivir, another common treatment, can cause gastrointestinal symptoms like nausea and vomiting, and has been associated with rare neuropsychiatric events, particularly in children. Zanamivir, administered via inhalation, may cause bronchospasm and is contraindicated in patients with respiratory disorders. Peramivir, given parenterally, commonly causes diarrhea and has rare but serious adverse effects like skin reactions and neuropsychiatric events.

What prior FDA approvals exist?

Baloxavir Marboxil, approved by the FDA, is an antiviral drug that inhibits the cap-dependent endonuclease activity of the influenza virus, thereby preventing viral replication. Other FDA-approved antiviral treatments for influenza include neuraminidase inhibitors such as oseltamivir (Tamiflu), zanamivir (Relenza), and peramivir (Rapivab), which work by blocking the neuraminidase enzyme, hindering the release of new viral particles from infected cells. These treatments are effective when administered shortly after the onset of symptoms.

What other types of research exist?

Promising novel alternatives to Baloxavir Marboxil for treating influenza include: 1) Combination therapy of Baloxavir acid with Ribavirin, which has shown a synergistic effect in inhibiting viral replication. 2) New antiviral agents under investigation, such as inhibitors of the main viral protease and entry/fusion inhibitors targeting the influenza virus spike protein. These alternatives offer potential enhanced efficacy and novel mechanisms of action against influenza.

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Anti-viral

Baloxavir Marboxil for Flu (Pebblestone Trial)

Phase 3

Recruiting

Research Sponsored by Hoffmann-La Roche

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Must not have

Participants with severe influenza virus infection requiring inpatient treatment.

Participants with concurrent (non-influenza) infections requiring systemic anti-microbial and/or anti-viral therapy at the pre-dose examinations.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to day 29

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing a flu medicine called baloxavir marboxil in children aged 1 to under 12 years. The medicine is given as a single dose and aims to stop the flu virus from spreading in the body, making the illness less severe and shorter. Baloxavir marboxil has been shown to reduce the duration of influenza symptoms by approximately one day.

Who is the study for?

This study is for children aged 1 to under 12 with confirmed influenza, who've shown symptoms within the last 48 hours and tested negative for COVID-19. They shouldn't have taken certain antiviral drugs recently or be severely immunocompromised. Girls who have started their periods are excluded.

What is being tested?

The trial tests how well Baloxavir Marboxil works before and after a single dose in treating young patients with the flu. It aims to monitor changes in susceptibility to this medication post-treatment.

What are the potential side effects?

While specific side effects aren't listed here, common ones from similar antiviral medications include nausea, diarrhea, headache, and potential allergic reactions.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am hospitalized due to a severe flu infection.

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I am not on any systemic anti-microbial or anti-viral therapy for infections other than the flu.

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I haven't had experimental flu antibody treatment recently.

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I am a woman who has started menstruating.

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I haven't taken flu medications like Tamiflu or Relenza in the last 30 days.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to day 29

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to day 29

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to day 29 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Number of Participants with Adverse Events and Serious Adverse Events

Side effects data

From 2020 Phase 4 trial • 481 Patients • NCT04141930

Diarrhea

Headache

100%

80%

60%

40%

20%

Study treatment Arm

Study Drug Eligible

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Baloxavir MarboxilExperimental Treatment1 Intervention

Participants will receive a single oral dose of baloxavir marboxil on Day 1 (based on body weight and age).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Baloxavir Marboxil

2019

Completed Phase 4

~8900

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Baloxavir marboxil works by inhibiting the cap-dependent endonuclease activity of the influenza virus, which is essential for viral mRNA synthesis, thereby preventing viral replication. In contrast, neuraminidase inhibitors like oseltamivir block the neuraminidase enzyme, stopping the release of new viral particles from infected cells. These mechanisms are crucial for influenza patients as they help reduce viral load, alleviate symptoms, and prevent complications by targeting different stages of the virus's life cycle.

Long-Term Maintenance of the Functional Changes Induced by Influenza A Virus and/or LPS in Human Endothelial ECV-304 Cell Sublines.Kinase inhibitor roscovitine as a PB2 cap-binding inhibitor against influenza a virus replication.Clinical efficacy and safety of baloxavir marboxil in the treatment of influenza: A systematic review and meta-analysis of randomized controlled trials.