Study looking at a new inhaled therapy shares encouraging results
What did the study look at?
The study looked at ETD001, a new inhaled therapy being developed by Enterprise Therapeutics Ltd. ETD001 is an ENaC blocker, which means it is designed to block channels that move salt into lung cells. This could help keep mucus in the airways moist and easier to clear.
This was a phase 2 study designed for people with CF who were not taking CFTR modulators. The study looked at whether the therapy could improve their lung function. Researchers also assessed whether ETD001 was safe and whether people were able to take it without significant problems.
54 adults with CF completed the study. They took part in CF centres in France, Germany, Italy and the UK.
The study had two parts. In part A, researchers monitored people for seven days to see whether the therapy was safe and caused any unwanted effects. People took the study treatment twice a day. In part B, everyone completed two 28-day treatment periods, with a 28-day break without study treatment in between. During one treatment period, they took ETD001 twice a day; during the other, they took a placebo. The order in which they received ETD001 and the placebo was decided at random.
What were the findings?
Results suggest that ETD001 improved lung function in people not taking modulators compared with placebo. Lung function changes were assessed using spirometry tests to measure ppFEV (percent-predicted forced expiratory volume) at the beginning and end of the treatment period. These results showed a 3.4% improvement in ppFEV compared to the placebo, a clinically meaningful benefit. These findings were supported by other lung function measures. The study also found that ETD001 was safe and generally caused few side effects for those taking it.
What’s next?
This is the first ENaC blocker therapy to show clinically meaningful improvements in lung function in people with CF compared to a placebo.
Enterprise Therapeutics Ltd are now intending to run a further phase 2 study to look at how effective ETD001 is when taken in combination with CFTR modulators. This will recruit people with CF who are currently taking CFTR modulators.
These studies are helping to push forward our understanding of new CF treatments.
It’s important that we continue to find new treatment options that benefit everyone with CF. While CFTR modulators have changed the lives of many people with CF, not everyone is able to take CFTR modulators and even among those who do, the benefits can vary significantly.
Through our Clinical Trials Accelerator Platform (CTAP), we support a range of new treatments being developed for CF. Find opportunities to take part in research on our Trials Tracker or by signing up for our CF research newsletter.
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