600 Participants Needed

Baloxavir Marboxil for Flu

(Pebblestone Trial)

Recruiting at 41 trial locations
RS
Overseen ByReference Study ID Number: CV44536 https://forpatients.roche.com/
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 3 JurisdictionsThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you have not taken certain flu medications like baloxavir marboxil, oseltamivir, or others within 30 days before joining. If you're on these, you might need to stop them before participating.

Is Baloxavir Marboxil safe for human use?

Baloxavir Marboxil, also known as Xofluza, has been approved in the United States for treating influenza, especially in those at high risk of complications, indicating it has undergone safety evaluations for this use.12345

How is the drug Baloxavir Marboxil unique for treating the flu?

Baloxavir Marboxil is unique because it is a first-in-class drug that works by inhibiting a specific protein in the flu virus, stopping it from replicating. It is taken as a single oral dose and can reduce the flu virus more quickly than some other treatments, like oseltamivir.678910

What is the purpose of this trial?

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.

Research Team

CT

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Eligibility Criteria

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.

Inclusion Criteria

You need to have a negative test for the coronavirus within 48 hours before the study screening.
I have symptoms like the flu and tested positive for it within the last day.
The time interval between the onset of influenza symptoms and the pre-dose examinations at screening is 48 hours or less

Exclusion Criteria

Treatment with an investigational therapy within 30 days or 5 half-lives, whichever is longer, prior to screening.
Known hypersensitivity to baloxavir marboxil or the drug product excipients.
I am hospitalized due to a severe flu infection.
See 5 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive a single oral dose of baloxavir marboxil on Day 1

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks
Up to Day 29

Treatment Details

Interventions

  • Baloxavir Marboxil
Trial Overview 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.
Participant Groups
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).

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

Find a Clinic Near You

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials
2,482
Recruited
1,107,000+
Headquarters
Basel, Switzerland
Known For
Precision medicine
Top Products
Avastin, Herceptin, Rituxan, Accu-Chek
Dr. Levi Garraway profile image

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker profile image

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University

Findings from Research

Lumacaftor-ivacaftor therapy has been shown to improve pulmonary function and reduce exacerbations in cystic fibrosis patients with the Phe-508del mutation, but it may also lead to side effects such as transaminitis and non-congenital cataracts.
The therapy can cause dyspnea in some patients, particularly at the start of treatment, and it has significant drug interaction risks due to lumacaftor's strong induction of CYP3A, which can affect the efficacy of other medications.
The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis.Talamo Guevara, M., McColley, SA.[2018]
In a study of 12 patients with severe cystic fibrosis (ppFEV1 <40), the combination therapy of lumacaftor/ivacaftor (LUM/IVA) led to a significant decline in lung function (median relative change of -19% at 2 hours), although most patients recovered by 1 month.
A high percentage (83.3%) of patients experienced non-severe respiratory-related adverse events within 24 hours of starting LUM/IVA, indicating that close monitoring for adverse effects is necessary in this population.
Immediate effects of lumacaftor/ivacaftor administration on lung function in patients with severe cystic fibrosis lung disease.Popowicz, N., Wood, J., Tai, A., et al.[2018]
In a study of 845 cystic fibrosis patients treated with lumacaftor-ivacaftor, those who continued the treatment experienced significant improvements in lung function (ppFEV1 increased by +3.67%) and nutritional status (body mass index increased by +0.73 kg/m2).
However, 18.2% of patients discontinued treatment, primarily due to respiratory and nonrespiratory adverse events, with discontinuation linked to older age and poorer lung function, indicating that while the drug is effective for many, it may pose risks for certain individuals.
Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis.Burgel, PR., Munck, A., Durieu, I., et al.[2020]

References

The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis. [2018]
Immediate effects of lumacaftor/ivacaftor administration on lung function in patients with severe cystic fibrosis lung disease. [2018]
Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis. [2020]
4.United Arab Emiratespubmed.ncbi.nlm.nih.gov
Cytochrome P450 3A4 Induction: Lumacaftor versus Ivacaftor Potentially Resulting in Significantly Reduced Plasma Concentration of Ivacaftor. [2020]
Influenza Therapeutics in Clinical Practice-Challenges and Recent Advances. [2023]
Cost-effectiveness of baloxavir marboxil compared to laninamivir for the treatment of influenza in Japan. [2021]
Baloxavir Marboxil: A Review in Acute Uncomplicated Influenza. [2021]
Association of baloxavir marboxil prescription with subsequent medical resource utilization among school-aged children with influenza. [2021]
Efficacy of baloxavir marboxil on household transmission of influenza infection. [2022]
Association Between Baloxavir Marboxil Prescription for Children with Influenza B Infections and Short-Term Healthcare Consumption in Japan During the 2018-2019 Influenza Season. [2022]
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