Clinical trial testing if an established blood cancer drug can avoid the need for ventilation in hospitalised COVID-19 patients over the age of 12

Research title

RUX-COVID: A single arm open-label clinical study to investigate the efficacy and safety of Ruxolitinib for the treatment of COVID-19 pneumonia


A two-stage Phase 2 trial to investigate if a JAK inhibitor can halt the progression of COVID-19 and avoid the need for a ventilator. Ruxolitinib, a JAK inhibitor currently used to treat myelofibrosis, a rare form of blood cancer, will be tested in 20 hospitalised patients in Ontario. Once results of Stage 1 are available, a further group of patients will be enrolled into Stage 2. The team will collaborate on the analysis of patients’ samples with Dr Shahram’s Kordasti’s team at King’s College, London who is also conducting a trial with ruxolitinib.

Research organisation

University Health Network, University of Toronto


  • Principal Investigator: Dr Steven Chan, University Health Network, University of Toronto, Princess Margaret Cancer Centre
  • Co-Principal Investigator: Dr Vikas Gupta, University Health Network, University of Toronto, Princess Margaret Cancer Centre

Potential of repurposed therapeutic for COVID-19 pandemic

In late stage COVID-19, hyperinflammation is seen. Studies have shown that in these severe cases of COVID-19, the levels of pro-inflammatory cytokines and biomarkers including interleukin 6 (IL-6) are highly elevated. In fact, similar pro-inflammatory cytokines are frequently overexpressed in autoimmune and inflammatory diseases. A pathway called JAK-STAT is responsible for controlling the level of these pro-inflammatory cytokines produced in the body. JAK inhibitors block the signals to produce more pro-inflammatory cytokines and therefore reduce the host’s inflammatory response. JAK inhibitors have been shown to be effective in a range of inflammatory diseases such as rheumatoid arthritis.

Ruxolitinib is a JAK inhibitor which is currently approved for myelofibrosis, a rare form of blood cancer. It is hypothesised that ruxolitinib will reduce JAK activity and ultimately prevent hyperinflammation in COVID-19 patients. Due to this promising link, there are a number of additional trials registered to test ruxolitinib and other JAK inhibitors for COVID-19.

In stage 1, 20 hospitalised COVID-19 patients above the age of 12 will be recruited to the trial. This lower age bracket has been approved due to trial data showing ruxolitinib appears to be tolerated and safe in children. The measure being used for the trial is the percentage of patients who progress to a severe stage of disease where they need mechanical ventilation and/or fraction of inspired oxygen of 60% or more. From reports from Canada and China, the current standard of care means that about 30% of hospitalised patients will progress to this severe stage. Therefore, if less than 25% of the patients receiving standard care and ruxolitinib progress, stage 1 has been successful. Stage 2 will run the same test on a further 44 patients. The manufacturer, Novartis, is supplying the oral pill ruxolitinib free of charge for the trial.