A study to evaluate a new ENaC blocker in adults with CF not using CFTR modulator therapies

A two-part study to assess the safety and effectiveness of repeat doses of a new inhaled ENaC blocker, ETD001, in people with cystic fibrosis (CF)

The aim of the study is to assess the safety and effectiveness of ETD001, a new therapy for people with CF to improve mucociliary clearance and lung function. If you can’t take CFTR modulator treatments, or you are not currently taking CFTR modulator treatments, you may be eligible to take part in this study. 

This is a new approach to nebulised medication that aids mucus clearance, to potentially improve lung function. It works in a different way to current nebulised medication. We need to conduct trials to see how effective this different mode of action is, to ensure we are offering people with CF the most effective meds for airway clearance and, if successful, providing options for treatments beyond what is currently offered. 

Taking part in this study will not prevent you from taking part in other trials in the future, including trials involving genetic therapies.

This study is split into 2 parts: part A and part B. 

Part A will assess if ETD001 is safe and tolerable in people with CF and how it affects the body when taken twice a day over 7 days. Eight people will take part in part A. They will be split into two groups randomly. Six people will receive ETD001 and 2 will receive a placebo. Part A of the study is now complete.

Part B will assess how well the study therapy works to improve lung function in people with CF when taken twice a day over 28 days. Part B has now started.

Thirty-two participants will be required take part in part B. Everyone will be asked to complete two 28-day treatment periods. During one treatment period, they will take ETD001 twice a day. During the other treatment period, they will take a placebo twice a day. The order in which people receive ETD001 and the placebo will be random.

Participants who have completed part A may also take part in part B.

Participants will be asked to attend the clinic for screening and then 6 visits on days 1, 14, 28, 57, 70 and 84. They will also be asked to attend a follow-up visit on day 105.