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

Chang AB 1 , Yerkovich ST 2 , Baines KJ 3 , Burr L 4 , Champion A 5 , Chatfield MD 6 Show all authors , Eg KP 7 , Goyal V 2 , Marsh RL 8 , McCallum GB 8 , McElrea M 2 , McPhail S 2 , Morgan LC 9 , Morris PS 8 , Nathan AM 7 , O'Farrell H 2 , Sanchez MO 10 , Parsons M 11 , Schultz A 12 , Torzillo PJ 13 , West NP 14 , Versteegh L 8 , Marchant JM 15 , Grimwood K 14

Affiliations 

  • 1 Department of Respiratory Medicine, Queensland Children's Hospital, South Brisbane, Queensland, Australia anne.chang@menzies.edu.au
  • 2 The Australian Centre for Health Services Innovation (AusHSI), Queensland University of Technology, Brisbane, Queensland, Australia
  • 3 School of Medicine and Public Health, The University of Newcastle, Newcastle, New South Wales, Australia
  • 4 Mater Health Services, South Brisbane, Queensland, Australia
  • 5 Department of Pharmacy, Queensland Children's Hospital, Brisbane, Queensland, Australia
  • 6 The University of Queensland, Brisbane, Queensland, Australia
  • 7 Department of Paediatrics, Universiti Malaya Faculty of Medicine, Kuala Lumpur, Malaysia
  • 8 Child and Maternal Health Division and andand NHMRC Centre for Research Excellence in Paediatric Bronchiectasis (AusBREATHE), Menzies School of Health Research, Darwin, Northern Territory, Australia
  • 9 Department of Respiratory Medicine, Concord Repatriation General Hospital, Concord, New South Wales, Australia
  • 10 Section of Pediatric Pulmonology, Institute of Pulmonary Medicine, St. Luke's Medical Center, Quezon City, Philippines
  • 11 Parent Advisory Group, Cough, Asthma and Airways Group, Queensland University of Technology Faculty of Health, Kelvin Grove, Queensland, Australia
  • 12 Department of Respiratory Medicine, Princess Margaret Hospital for Children, Perth, Western Australia, Australia
  • 13 Respiratory Medicine, Royal Prince Alfred Hospital, Camperdown, New South Wales, Australia
  • 14 School of Medicine and Dentistry, Griffith University Griffith Health, Gold Coast, Queensland, Australia
  • 15 Department of Respiratory Medicine, Queensland Children's Hospital, South Brisbane, Queensland, Australia
BMJ Open Respir Res, 2024 May 07;11(1).
PMID: 38719503 DOI: 10.1136/bmjresp-2023-002216

Abstract

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.

TRIAL REGISTRATION NUMBER: ACTRN12621000315819.

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.

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