Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial
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...
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2024
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R Medicine Chang, Anne B. Yerkovich, Stephanie T. Baines, Katherine J. Burr, Lucy Champion, Anita Chatfield, Mark D. Eg, Kah Peng Goyal, Vikas Marsh, Robyn L. McCallum, Gabrielle B. McElrea, Margaret McPhail, Steven Morgan, Lucy C. Morris, Peter S. Nathan, Anne Marie O'Farrell, Hannah Sanchez, Marion O. Parsons, Marianne Schultz, Andre Torzillo, Paul J. West, Nicholas P. Versteegh, Lesley Marchant, Julie M. Grimwood, Keith Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial |
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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.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.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. |
| format |
Article |
| author |
Chang, Anne B. Yerkovich, Stephanie T. Baines, Katherine J. Burr, Lucy Champion, Anita Chatfield, Mark D. Eg, Kah Peng Goyal, Vikas Marsh, Robyn L. McCallum, Gabrielle B. McElrea, Margaret McPhail, Steven Morgan, Lucy C. Morris, Peter S. Nathan, Anne Marie O'Farrell, Hannah Sanchez, Marion O. Parsons, Marianne Schultz, Andre Torzillo, Paul J. West, Nicholas P. Versteegh, Lesley Marchant, Julie M. Grimwood, Keith |
| author_facet |
Chang, Anne B. Yerkovich, Stephanie T. Baines, Katherine J. Burr, Lucy Champion, Anita Chatfield, Mark D. Eg, Kah Peng Goyal, Vikas Marsh, Robyn L. McCallum, Gabrielle B. McElrea, Margaret McPhail, Steven Morgan, Lucy C. Morris, Peter S. Nathan, Anne Marie O'Farrell, Hannah Sanchez, Marion O. Parsons, Marianne Schultz, Andre Torzillo, Paul J. West, Nicholas P. Versteegh, Lesley Marchant, Julie M. Grimwood, Keith |
| author_sort |
Chang, Anne B. |
| title |
Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial |
| title_short |
Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial |
| title_full |
Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial |
| title_fullStr |
Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial |
| title_full_unstemmed |
Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial |
| title_sort |
erdosteine in children and adults with bronchiectasis (better trial): study protocol for a multicentre, double-blind, randomised controlled trial |
| publisher |
BMJ Publishing Group |
| publishDate |
2024 |
| url |
http://eprints.um.edu.my/45258/ https://doi.org/10.1136/bmjresp-2023-002216 |
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1811682110031790080 |
| spelling |
my.um.eprints.452582024-09-30T04:56:38Z http://eprints.um.edu.my/45258/ Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial Chang, Anne B. Yerkovich, Stephanie T. Baines, Katherine J. Burr, Lucy Champion, Anita Chatfield, Mark D. Eg, Kah Peng Goyal, Vikas Marsh, Robyn L. McCallum, Gabrielle B. McElrea, Margaret McPhail, Steven Morgan, Lucy C. Morris, Peter S. Nathan, Anne Marie O'Farrell, Hannah Sanchez, Marion O. Parsons, Marianne Schultz, Andre Torzillo, Paul J. West, Nicholas P. Versteegh, Lesley Marchant, Julie M. Grimwood, Keith R Medicine 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.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.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. BMJ Publishing Group 2024-05 Article PeerReviewed Chang, Anne B. and Yerkovich, Stephanie T. and Baines, Katherine J. and Burr, Lucy and Champion, Anita and Chatfield, Mark D. and Eg, Kah Peng and Goyal, Vikas and Marsh, Robyn L. and McCallum, Gabrielle B. and McElrea, Margaret and McPhail, Steven and Morgan, Lucy C. and Morris, Peter S. and Nathan, Anne Marie and O'Farrell, Hannah and Sanchez, Marion O. and Parsons, Marianne and Schultz, Andre and Torzillo, Paul J. and West, Nicholas P. and Versteegh, Lesley and Marchant, Julie M. and Grimwood, Keith (2024) Erdosteine in children and adults with bronchiectasis (BETTER trial): study protocol for a multicentre, double-blind, randomised controlled trial. BMJ Open Respiratory Research, 11 (1). e002216. ISSN 2052-4439, DOI https://doi.org/10.1136/bmjresp-2023-002216 <https://doi.org/10.1136/bmjresp-2023-002216>. https://doi.org/10.1136/bmjresp-2023-002216 10.1136/bmjresp-2023-002216 |
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13.211869 |