Clinical Trials

Numerous clinical trials evaluate baloxavir marboxil across various viral infections and respiratory conditions, primarily targeting influenza and influenza A, alongside pneumonia, COVID-19, and acute exacerbations of chronic obstructive pulmonary disease. Spanning Phase 2 through Phase 4, these protocols encompass completed pediatric and adult investigations as well as actively recruiting and non-recruiting studies. The trial landscape is sponsored by pharmaceutical companies, such as Hoffmann-La Roche and Shionogi, and academic institutions, including the University of Oxford.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT04381936 RECRUITING
Pneumonia
University of Oxford
 2020-03-19 PHASE3
NCT04712539 RECRUITING
Hematopoietic and Lymphoid Cell Neoplasm; Influenza
M.D. Anderson Cancer Center
 2021-10-11 PHASE2
NCT06094010 RECRUITING
Influenza
Hoffmann-La Roche
 2023-11-22 PHASE3
NCT06653569 RECRUITING
Influenza Disease; Flu
Dre Pauline Vetter
 2024-12-13 PHASE3
NCT02735707 RECRUITING
Community-acquired Pneumonia, Influenza, COVID-19
UMC Utrecht
 2016-04-11 PHASE3
NCT06774859 COMPLETED
Influenza
Hoffmann-La Roche
 2024-10-27 PHASE3
NCT06161454 COMPLETED
Infection, Coronavirus; Infections; Influenza; Transplant; Infection Viral
Children's Hospital of Philadelphia
 2023-12-14 PHASE4
NCT06762587 NOT_YET_RECRUITING
Influenza; Influenza Prophylaxis; Nosocomial Infection
Capital Medical University
 2025-01-05
NCT06507813 COMPLETED
Influenza Type A; Influenza Type B
Jiaxing AnDiCon Biotech Co.,Ltd
 2024-08-19 PHASE3
NCT06205641 UNKNOWN
Influenza A
Capital Medical University
 2024-01
NCT03969212 COMPLETED
Influenza
Hoffmann-La Roche
 2019-10-10 PHASE3
NCT05170009 TERMINATED
Influenza
Weill Medical College of Cornell University
 2022-04-22 PHASE2; PHASE3
NCT06094010 Recruiting
Influenza
Hoffmann-La Roche
 2023-11-22 Phase 3
NCT03653364 COMPLETED
Influenza
Hoffmann-La Roche
 2019-01-23 PHASE3
NCT04141930 COMPLETED
Influenza; Respiratory Viral Infection
University of Washington
 2019-11-01 PHASE4
NCT03684044 COMPLETED
Influenza
Hoffmann-La Roche
 2019-01-08 PHASE3
NCT03959332 COMPLETED
Healthy Volunteer
Hoffmann-La Roche
 2019-06-19
NCT03629184 COMPLETED
Influenza
Hoffmann-La Roche
 2018-11-20 PHASE3
NCT03653364 Completed
Influenza
Hoffmann-La Roche
 2019-01-23 Phase 3
NCT03629184 Completed
Influenza
Hoffmann-La Roche
 2018-11-20 Phase 3
NCT02949011 COMPLETED
Influenza
Shionogi
 2017-01-11 PHASE3
NCT02954354 COMPLETED
Influenza
Shionogi
 2016-12-08 PHASE3

(data from https://clinicaltrials.gov, updated on 2026-04-21)

Check the Baloxavir marboxil product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Baloxavir marboxil functions as a prodrug that converts to its active metabolite to selectively inhibit the polymerase acidic cap-dependent endonuclease, thereby blocking the cap-snatching process required for viral messenger RNA synthesis. By halting viral gene expression and intracellular replication, this mechanism reduces viral shedding and alleviates symptoms in patients suffering from influenza and other viral respiratory infections.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.