Article ; Online: Safety and efficacy of the epithelial sodium channel blocker idrevloride in people with primary ciliary dyskinesia (CLEAN-PCD): a multinational, phase 2, randomised, double-blind, placebo-controlled crossover trial.
The Lancet. Respiratory medicine
2023 Volume 12, Issue 1, Page(s) 21–33
Abstract: Background: Mucociliary clearance is dysfunctional in people with primary ciliary dyskinesia, resulting in the accumulation of dehydrated mucus in the airways that is difficult to clear. We undertook a study to assess the benefit on lung function of ... ...
| Abstract | Background: Mucociliary clearance is dysfunctional in people with primary ciliary dyskinesia, resulting in the accumulation of dehydrated mucus in the airways that is difficult to clear. We undertook a study to assess the benefit on lung function of treatment with a nebulised epithelial sodium channel (ENaC) blocker, idrevloride, with or without hypertonic saline, in people with primary ciliary dyskinesia. Methods: The CLEAN-PCD trial was a phase 2, randomised, double-blind, placebo-controlled crossover trial conducted at 32 tertiary adult and paediatric care centres and university hospitals in Canada, Denmark, Germany, Italy, the Netherlands, Poland, the UK, and the USA. People with a confirmed diagnosis of primary ciliary dyskinesia, aged 12 years or older, with a percentage of predicted FEV Findings: Between Sep 14, 2016, and May 31, 2018, 216 patients were screened and 123 were randomly assigned to one of four crossover sequences. Across the two treatment periods, treatment with idrevloride in hypertonic saline was initiated in 80 patients and completed in 78 patients (all 78 had data available and were included in the analysis); hypertonic saline initiated in 81 patients and completed in 76 patients (75 had data available and were included in the analysis); idrevloride initiated in 37 patients and completed in 35 patients (34 had data available and were included in the analysis); and placebo initiated in 36 patients and completed in 34 patients (all 34 had data available and were included in the analysis). Greater absolute increases in ppFEV Interpretation: In this phase 2 crossover study, idrevloride in hypertonic saline was safe and associated with improved lung function over a 28-day period in people with primary ciliary dyskinesia compared with hypertonic saline alone. Larger, longer clinical studies are warranted to explore the potential benefits of idrevloride in combination with hypertonic saline in people with primary ciliary dyskinesia. Funding: Parion Sciences, under agreement with Vertex Pharmaceuticals. |
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| MeSH term(s) | Adult ; Child ; Humans ; Cross-Over Studies ; Epithelial Sodium Channel Blockers ; Treatment Outcome ; Mucociliary Clearance ; Ciliary Motility Disorders ; Double-Blind Method |
| Chemical Substances | Epithelial Sodium Channel Blockers |
| Language | English |
| Publishing date | 2023-08-31 |
| Publishing country | England |
| Document type | Randomized Controlled Trial ; Clinical Trial, Phase II ; Journal Article |
| ZDB-ID | 2686754-0 |
| ISSN | 2213-2619 ; 2213-2600 |
| ISSN (online) | 2213-2619 |
| ISSN | 2213-2600 |
| DOI | 10.1016/S2213-2600(23)00226-6 |
| Database | MEDical Literature Analysis and Retrieval System OnLINE |
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