RecruitingPhase 3Phase 4ACTRN12621000315819

BronchiEctasis Trial Testing ERdosteine (BETTER)

Evaluating the effect of erdosteine on respiratory exacerbation rate of children and adults with bronchiectasis - a double-blind, randomised controlled trial


Sponsor

Queensland University of Technology

Enrollment

205 participants

Start Date

May 27, 2021

Study Type

Interventional

Conditions

Summary

Bronchiectasis is a commonly seen chronic lung disease in our inpatient and outpatient services. The unmet needs of people with bronchiectasis are huge, with relatively few randomised controlled trials (RCTs) and evidence-based interventions. This RCT aims to improve the outcomes of children and young adults with bronchiectasis. It will examine the benefits (or otherwise) of a novel medication, erdosteine. Erdosteine’s effects include (a) mucolytic action, modulation of mucus production and increasing muco-ciliary transport; with (b) antioxidant; (c) airway anti-inflammatory and; (d) bacterial anti-adhesion properties. This RCT will answer: In children and adults aged <49 years with bronchiectasis, does 12 months treatment with erdosteine, compared to placebo, (i) reduce acute respiratory exacerbations and/or (ii) improve quality of life (QoL). The RCT will also determine the cost-effectiveness of the treatment.


Eligibility

Sex: Both males and femalesMin Age: 2 YearssMax Age: 49 Yearss

Plain Language Summary

Simplified for easier understanding

Bronchiectasis is a chronic lung disease in which the airways become permanently damaged and widened, making it hard to clear mucus and leading to repeated chest infections. It is particularly challenging for children and young people, where evidence-based treatments remain limited. The BETTER trial is testing a medication called erdosteine, which has several potentially beneficial effects: it helps thin and clear mucus, reduces inflammation in the airways, has antioxidant properties, and may even make it harder for bacteria to stick to the airway walls. This trial is open to people aged 2 to 49 years with bronchiectasis who have had at least two chest exacerbations in the past 18 months. Participants will take either erdosteine or a placebo for 12 months, and the study will measure whether the medication reduces the number of chest infections and improves quality of life. Participants will be followed up for 15 months in total. People with cystic fibrosis, significant liver or kidney disease, or those who are pregnant or planning pregnancy are not eligible. This study is particularly important because there are very few proven treatments for non-cystic fibrosis bronchiectasis in children, and it also includes a cost-effectiveness analysis to determine value for money.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

Erdosteine: Oral twice daily doses for 12 months [<15 kg = 2.5mls/dose; 15-19 kg = 5mls/dose; 20-30 kg = 7.5ml/dose (or one capsule in children who can take capsules); >30 kg = 10ml/dose (or one capsu

Erdosteine: Oral twice daily doses for 12 months [<15 kg = 2.5mls/dose; 15-19 kg = 5mls/dose; 20-30 kg = 7.5ml/dose (or one capsule in children who can take capsules); >30 kg = 10ml/dose (or one capsule in participants who can take capsules)]. The formulation for liquid is powder for suspension. Adherence will be monitored by return of bottles or capsule packaging. In a subset of participants, we will also evaluate if inflammatory and/or microbial biomarkers will identify pathways and/or predict those at greater risk of recurrent respiratory exacerbations. These outcomes will not be reported in the RCT. Participants for this component will be based on whether they consent to these additional procedures (sputum and blood). Where possible, bloods and sputum will be taken at baseline and at commencement of an exacerbation.


Locations(8)

Queensland Children's Hospital - South Brisbane

QLD, Australia

Gold Coast University Hospital - Southport

QLD, Australia

Royal Darwin Hospital - Tiwi

QLD, Australia

Mater Adult Hospital - South Brisbane

QLD, Australia

Concord Repatriation Hospital - Concord

QLD, Australia

Royal Perth Hospital - Perth

QLD, Australia

Kuala Lumpur, Malaysia

Quezon City, Philippines

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ACTRN12621000315819


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