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Acronym
REPEAT
Project Title
REPEAT Study: Improving outcomes of children and young adults with primary ciliary dyskinesia: a multi-centre, double blind, double-dummy, 2x2 partial factorial, randomised controlled trial
Principal Investigator
Start Date
August 25, 2020
Investigators
Project Web Site
Keywords
Primary ciliary dyskinesia
Project Type
Interventional/Clinical trials research
Location
Region
Keywords

Primary ciliary dyski...

Description
Primary ciliary dyskinesia (PCD), is a rare, incurable, progressive, serious disorder with a large
unmet need in both the diagnosis & treatment. To date, all currently used therapies are unproven.
Our parallel, multicentre, 2x2 factorial design, double-blinded, double-dummy, superiority
randomised controlled trial (concealed 1:1:1:1 allocation) will test the benefits of a currently used
(but unapproved for long term use in PCD) antibiotic (azithromycin) and a novel mucolytic
(erdosteine). Our primary question is: among children/young adults with PCD, does azithromycin or
erdosteine reduce acute respiratory exacerbations during 12 months of treatment?

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