Safety and Efficacy of Dupilumab for the Treatment of Hospitalized Patients With Moderate to Severe Coronavirus Disease 2019: A Phase 2a Trial

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Abstract

Background

Based on studies implicating the type 2 cytokine interleukin 13 (IL-13) as a potential contributor to critical coronavirus disease 2019 (COVID-19), this trial was designed as an early phase 2 study to assess dupilumab, a monoclonal antibody that blocks IL-13 and interleukin 4 signaling, for treatment of inpatients with COVID-19.

Methods

We conducted a phase 2a randomized, double-blind, placebo-controlled trial (NCT04920916) to assess the safety and efficacy of dupilumab plus standard of care vs placebo plus standard of care in mitigating respiratory failure and death in those hospitalized with COVID-19.

Results

Forty eligible subjects were enrolled from June to November of 2021. There was no statistically significant difference in adverse events nor in the primary endpoint of ventilator-free survival at day 28 between study arms. However, for the secondary endpoint of mortality at day 60, there were 2 deaths in the dupilumab group compared with 5 deaths in the placebo group (60-day survival: 89.5% vs 76.2%; adjusted hazard ratio [HR], 0.05 [95% confidence interval {CI}, .004–.72]; P = .03). Among subjects who were not in the intensive care unit (ICU) at randomization, 3 subjects in the dupilumab arm were admitted to the ICU compared to 6 in the placebo arm (17.7% vs 37.5%; adjusted HR, 0.44 [95% CI, .09–2.09]; P = .30). Last, we found evidence of type 2 signaling blockade in the dupilumab group through analysis of immune biomarkers over time.

Conclusions

Although the primary outcome of day 28 ventilator-free survival was not reached, adverse events were not observed and survival was higher in the dupilumab group by day 60.

Clinical Trials Registration

NCT04920916.

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  1. SciScore for 10.1101/2022.03.30.22273194: (What is this?)

    Please note, not all rigor criteria are appropriate for all manuscripts.

    Table 1: Rigor

    EthicsIRB: It was approved by the University of Virginia Institutional Review Board (IRB) in June 2021 (NCT04920916).
    Sex as a biological variablenot detected.
    RandomizationDesign: This was a randomized, double-blind, placebo-controlled trial designed to assess the safety and efficacy of dupilumab use in 40 hospitalized patients with moderate to severe COVID-19 infection.
    Blindingnot detected.
    Power AnalysisWith a pre-selected sample size of 40 patients and alpha=0.1 (one sided), we would be able to detect a difference of 17.7% in the proportion of subjects alive and free of mechanical ventilation at 28 days with 75% power.
    Cell Line AuthenticationAuthentication: Ferritin, CRP and IgE levels were measured at the University of Virginia Clinical Laboratories while other biomarkers were measured by multiplex immunoassays or ELISAs depending on the analyte.

    Table 2: Resources

    Software and Algorithms
    SentencesResources
    Additional type 2 inflammatory markers including TARC (CCL17), YKL40, eotaxin 3 (CCL26), arginase1 (Arg1), hyaluronan, soluble ST2 and total serum immunoglobulin E (IgE) were also measured.
    TARC
    suggested: None

    Results from OddPub: We did not detect open data. We also did not detect open code. Researchers are encouraged to share open data when possible (see Nature blog).


    Results from LimitationRecognizer: We detected the following sentences addressing limitations in the study:
    The study had several limitations. These included the lack of achievement of the primary endpoint of proportion of patients alive and free of mechanical ventilation at day 28, and the wide confidence intervals in the survival benefit of dupilumab at day 60. Additional limitations included unequal gender distribution between groups, and a higher-than-expected overall mortality rate. The study also had several notable strengths including having as a foundation the preclinical data on the mechanism of disease exacerbation by IL-13 in COVID-19, originality in the study of type 2 immune inhibition, the use of a prospective placebo-controlled randomized and double-blind design, and demonstration of the safety of dupilumab. Importantly, there was evidence for mortality reduction and reduced ICU escalation with dupilumab use as we had predicted from animal models and retrospective human studies, despite sample size limitations. In light of the ongoing need for additional therapies for COVID-19 associated respiratory failure and the modest clinical benefits seen with other anti-viral and immunotherapies currently being used, the results of this study advance dupilumab as a promising treatment option for those hospitalized with COVID-19.

    Results from TrialIdentifier: We found the following clinical trial numbers in your paper:

    IdentifierStatusTitle
    NCT04920916CompletedSafety and Efficacy of Dupilumab for Treatment of Hospitaliz…


    Results from Barzooka: We did not find any issues relating to the usage of bar graphs.


    Results from JetFighter: We did not find any issues relating to colormaps.


    Results from rtransparent:
    • Thank you for including a conflict of interest statement. Authors are encouraged to include this statement when submitting to a journal.
    • Thank you for including a funding statement. Authors are encouraged to include this statement when submitting to a journal.
    • No protocol registration statement was detected.

    Results from scite Reference Check: We found no unreliable references.


    About SciScore

    SciScore is an automated tool that is designed to assist expert reviewers by finding and presenting formulaic information scattered throughout a paper in a standard, easy to digest format. SciScore checks for the presence and correctness of RRIDs (research resource identifiers), and for rigor criteria such as sex and investigator blinding. For details on the theoretical underpinning of rigor criteria and the tools shown here, including references cited, please follow this link.