Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia
Research type
Research Study
Full title
A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block Crossover Study to Evaluate the Safety and Efficacy of VX-371 Solution for Inhalation in Subjects with Primary Ciliary Dyskinesia
IRAS ID
207336
Contact name
Anita Woodring
Contact email
Sponsor organisation
Parion Sciences, Incorporated
Eudract number
2015-004917-26
Duration of Study in the UK
2 years, 1 months, 1 days
Research summary
This study is a Phase2a, double-blind (neither the site staff not the participant would know if the participant is assigned to study medciation or its placebo equivalent), placebo-controlled study to determine the safety and efficacy of VX-371, an inhalational solution, in participants with Primary ciliary dyskinesia (PCD). Approximately 150 participants, aged 12 years or older, with a confirmed diagnosis of PCD will the randomised to one of the four treatment sequences [VX-371 in 4.2% Hypertonic Saline (HS), 4.2% HS, VX-371 in 0.17% saline (placebo) and Placebo (0.17% saline)]. Each of these sequences will compose of Treatment period 1, Washout, Treatment period 2. The study includes a screening period, treatment period and safety follow up.
The patients with PCD have hyperconcentrated (thick) mucus, resulting in poor cough clearance. VX-371 is expected to restore hydration of the mucus, thus resulting in improved cough clearance and combining it with HS is expected to further enhance the effectiveness of cough clearance.
REC name
London - Chelsea Research Ethics Committee
REC reference
16/LO/1424
Date of REC Opinion
24 Oct 2016
REC opinion
Further Information Favourable Opinion