Erdosteine in children and adults with bronchiectasis (BETTER trial): Study protocol for a multicentre, double-blind, randomised controlled trial

Anne B. Chang, Stephanie T. Yerkovich, Katherine J. Baines, Lucy Burr, Anita Champion, Mark D. Chatfield, Kah P. Eg, Vikas Goyal, Robyn L. Marsh, Gabrielle B. McCallum, Margaret McElrea, Steven McPhail, Lucy C. Morgan, Peter S. Morris, Anne M. Nathan, Hannah O'Farrell, Marion O. Sanchez, Marianne Parsons, André Schultz, Paul J. TorzilloNicholas P. West, Lesley Versteegh, Julie M. Marchant, Keith Grimwood

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Introduction Bronchiectasis is a worldwide chronic lung disorder where exacerbations are common. It affects people of all ages, but especially Indigenous populations in high-income nations. Despite being a major contributor to chronic lung disease, there are no licensed therapies for bronchiectasis and there remain relatively few randomised controlled trials (RCTs) conducted in children and adults. Our RCT will address some of these unmet needs by evaluating whether the novel mucoactive agent, erdosteine, has a therapeutic role in children and adults with bronchiectasis. Our primary aim is to determine in children and adults aged 2–49 years with bronchiectasis whether regular erdosteine over a 12-month period reduces acute respiratory exacerbations compared with placebo. Our primary hypothesis is that people with bronchiectasis who regularly use erdosteine will have fewer exacerbations than those receiving placebo. Our secondary aims are to determine the effect of the trial medications on quality of life (QoL) and other clinical outcomes (exacerbation duration, time-to-next exacerbation, hospitalisations, lung function, adverse events). We will also assess the cost-effectiveness of the intervention.

Methods and analysis We are undertaking an international multicentre, double-blind, placebo-RCT to evaluate whether 12 months of erdosteine is beneficial for children and adults with bronchiectasis. We will recruit 194 children and adults with bronchiectasis to a parallel, superiority RCT at eight sites across Australia, Malaysia and Philippines. Our primary endpoint is the rate of exacerbations over 12 months. Our main secondary outcomes are QoL, exacerbation duration, time-to-next exacerbation, hospitalisations and lung function. 

Ethics and dissemination The Human Research Ethics Committees (HREC) of Children’s Health Queensland (for all Australian sites), University of Malaya Medical Centre (Malaysia) and St. Luke’s Medical Centre (Philippines) approved the study. We will publish the results and share the outcomes with the academic and medical community, funding and relevant patient organisations.

Original languageEnglish
Article numbere002216
Pages (from-to)1-10
Number of pages10
JournalBMJ Open Respiratory Research
Issue number1
Publication statusPublished - 7 May 2024

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© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ.


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