Hematopoietic and Lymphoid Cell NeoplasmPhase 2NCT04712539
Baloxavir and Oseltamivir for the Treatment of Severe Influenza Infection in Immunocompromised Patients
A Phase 2 study testing Baloxavir Marboxil and Oseltamivir in people with Hematopoietic and Lymphoid Cell Neoplasm and Influenza, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This phase II trial studies the effect of baloxavir in combination with oseltamivir in treating severe influenza infection in patients who have previously received a hematopoietic (blood) stem cell transplant or have a hematological malignancy. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hematopoietic and Lymphoid Cell Neoplasm, Influenza
- What is being tested?
- Baloxavir Marboxil and Oseltamivir
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Oct 2021to 28 Feb 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Hematopoeitic cell transplant recipients OR hematological malignancy patients
- Diagnosed with influenza ⱡ
- Evidence of LRTI* or high risk upper respiratory tract infection (URTI)**
- ⱡ A positive multiplex PCR for influenza is required to confirm a diagnosis of influenza infection.
- LRTI will be defined as influenza cases that have evidence of disease below the level of the trachea on either imaging only (possible LRTI), imaging and microbiological evidence of lower airway disease with a bronchoscopy (probable LRTD) or pathological evidence of disease via biopsy (proven LRTI).
- High risk URI will be defined as those cases of influenza that do not have microbiological nor radiological evidence of LRTI, yet they have an immunodeficiency scoring index (ISI) of 3 or greater as defined by Shah D et al (19) for HCT recipients or severe neutropenia (ANC ≤500 cells/ml) and/or lymphopenia (ALC ≤200 cells/ml) for HM patients.
Who cannot join
- Patient requires mechanical ventilation at time of enrollment
- Patient is younger than the age of 12 years old
- The patient is unable to tolerate oral therapy
- The patient is pregnant at screening ( Positive serum β-HCG (beta-human chorionic gonadotropin) test for women of child-bearing potential).
- The patient is on a prohibited medication. These include Influenza antiviral drugs with the exception of oseltamivir and baloxavir (such as peramivir, laninamivir, zanamivir, rimantadine, umifenovir or amantadine) and herbal therapies.
- The patient is unable to consent will be excluded
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
M D Anderson Cancer Center
Houston, Texas, 77030
Roy F. Chemaly, MD,MPH, 713-792-6830, rfchemaly@mdanderson.org
Roy F. Chemaly, MD,MPH
About this study
Official title: Efficacy of Combination Baloxovir and Oseltamivir Therapy in Influenza Infected Immunocompromised Hosts
This phase II trial studies the effect of baloxavir in combination with oseltamivir in treating severe influenza infection in patients who have previously received a hematopoietic (blood) stem cell transplant or have a hematological malignancy. Baloxavir is an antiviral drug that inhibits the growth of influenza virus, reduces viral load and prevents further influenza infection. Osetamivir is an antiviral drug that blocks enzymes on the surfaces of influenza viruses, interfering with cell release of complete viral particles. Giving baloxavir in combination with oseltamivir may shorten or decrease the intensity of influenza infection compared to oseltamivir alone.
Read the detailed description
PRIMARY OBJECTIVE:
- To compare the efficacy of baloxavir marboxil (baloxavir) in combination with oseltamivir to oseltamivir monotherapy as measured by changes in influenza viral loads at day 1 from baseline for treatment of severe influenza infections in immunocompromised hosts (such as hematopoietic cell transplant [HCT] recipients and hematological malignancy [HM] patients) and compare the main clinical outcome, complicated hospital stay between the intervention arm and control arm.
SECONDARY OBJECTIVES:
- To compare the efficacy of baloxavir in combination with oseltamivir to oseltamivir monotherapy as measured by changes in influenza viral loads at day 3, 7, 14 and 30 from baseline.
II. To measure the incidence of baloxavir and oseltamivir resistance, development of lower respiratory tract infections (LRTI), oxygen requirement, respiratory failure, changes in microbiome of the upper airway, length of hospital stay and all-cause mortality at day 30 while on baloxavir and/or oseltamivir in these immunocompromised hosts.
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM I: Patients receive oseltamivir orally (PO) twice daily (BID) for up to 10 days and baloxavir marboxil PO every 72 hours for a total of 3 doses in the absence of disease progression or unacceptable toxicity.
ARM II: Patients receive oseltamivir PO BID for up to 10 days in the absence of disease progression or unacceptable toxicity.
After completion of study, patients are followed up at 30 days.
- Being tested
- Baloxavir Marboxil, Oseltamivir
- Medical subject headings
- Hematologic Neoplasms, Influenza, Human
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Baloxavir MarboxilDrug
Also called BXM, Xofluza
Given PO
OseltamivirDrug
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Arm I (oseltamivir, baloxavir marboxil) | Experimental | Patients receive oseltamivir PO BID for up to 10 days and baloxavir marboxil PO every 72 hours for a total of 3 doses in the absence of disease progression or unacceptable toxicity. Receives: Drug: Baloxavir Marboxil; Drug: Oseltamivir |
| Arm II (oseltamivir) | Active comparator | Patients receive oseltamivir PO BID for up to 10 days in the absence of disease progression or unacceptable toxicity. Receives: Drug: Oseltamivir |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in viral loads
Will be measured via repeat nasopharyngeal swabs at each follow up on day 0, 1, 3, 7, 14 and 30 for influenza quantification.
Measured: On day 0, 1, 3, 7, 14, and 30
Incidence of complicated hospital stay
Defined as a hospital admission that was either prolonged (greater than 7 days), requiring intensive care unit level of care or death at day 30 as a result of influenza infection.
Measured: Up to 30 days
Secondary outcomes (7)
Rate of resistance to antiviral agents
Will compare variables between the interventional and control groups using Fischer's exact test or Wilcoxon rank sum test when appropriate.
Measured: Up to 30 days
Progression to lower respiratory tract infections
Will compare variables between the interventional and control groups using Fischer's exact test or Wilcoxon rank sum test when appropriate.
Measured: Up to 30 days
Length of hospital stay
Will compare variables between the interventional and control groups using Fischer's exact test or Wilcoxon rank sum test when appropriate.
Measured: Up to 30 days
Oxygen requirement
Will compare variables between the interventional and control groups using Fischer's exact test or Wilcoxon rank sum test when appropriate.
Measured: Up to 30 days
Rate of respiratory failure
Will compare variables between the interventional and control groups using Fischer's exact test or Wilcoxon rank sum test when appropriate.
Measured: Up to 30 days
30-day mortality
Will compare variables between the interventional and control groups using Fischer's exact test or Wilcoxon rank sum test when appropriate.
Measured: At 30 days
Changes in microbiome diversity
Analysis of alpha and beta diversity of microbiome will be assessed using Agile Toolkit for Incisive Microbial Analyses. Using Shannon index, we will quantify the alpha diversity of the microbiome. Changes in microbiome diversity will be made by comparing alpha diversity at each time point of sample collection (days 0, 1, 3, 7, 14 and 30).
Measured: On day 0, 1, 3, 7, 14, and 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2021
- Study start11 Oct 2021
- Primary completion28 Feb 2028 (estimated)
- Study completion28 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematopoietic and Lymphoid Cell NeoplasmStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Roy F Chemaly, MD,MPH, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04712539
- Sponsor's protocol ID
- 2020-0919
- Other IDs
- NCI-2020-13918, 2020-0919
- Status last verified
- Jul 2026
- First submitted
- 13 Jan 2021
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Vetter P, Cabecinhas ARG, Schibler M, Kaiser L, Cruz-Fauquex K, Chalandon Y, Masouridi-Levrat S, Neofytos D. Use of baloxavir as adjunctive antiviral therapy to neuraminidase inhibitors in severely immunocompromised individuals infected with influenza. Antimicrob Agents Chemother. 2026 May 6;70(5):e0165925. doi: 10.1128/aac.01659-25. Epub 2026 Mar 27.
PubMed 41891860