Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia

Phase 2 Completed 123 enrolled Parion Sciences Vertex Pharmaceuticals Incorporated · collab

RandomizedCrossoverTriple-blindTreatment

Summary

To evaluate the safety and efficacy of treatment with VX-371 with and without ivacaftor, and the effect of VX-371 with and without ivacaftor on quality of life (QOL) in subjects with primary ciliary dyskinesia (PCD).

Timeline

Start: 2016-08
Primary completion: 2018-11-20
Completion: 2018-11-20

Outcome

Met primary endpoint

registry analysis (superiority test); Part A: VX-371 in HS vs Part A: HS; p = 0.0437; Least Square (LS) Mean difference 1.519 (95% CI 0.044 to 2.995); Mixed-effects Model NCT02871778 ↗

paper Greater absolute increases in ppFEV1 from baseline to 28 days of treatment were seen with idrevloride in hypertonic saline PMID 37660715 ↗

Publications

Ringshausen FC, Shapiro AJ, Nielsen KG, Mazurek H, Pifferi M, Donn KH, van der Eerden MM, Loebinger MR, Zariwala MA, Leigh MW, Knowles MR, Ferkol TW; CLEAN-PCD investigators and study team. 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. Lancet Respir Med. 2024 Jan;12(1):21-33. doi: 10.1016/S2213-2600(23)00226-6. Epub 2023 Aug 31.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Hypertonic saline	Unknown	3 ml	Inhaled
Subject	Hypertonic saline	Unknown	4.2 %	Inhaled
Subject	IDREVLORIDE	Small molecule	85 ug	Inhaled
Subject	Ivacaftor	Other / unclassified	150 mg	Oral