Chang 2013.
| Trial name or title | BEST‐2 Trial |
| Methods |
Aims: is daily oral azithromycin non‐inferior (within a 20% margin) to oral amoxicillin‐clavulanate in achieving resolution of exacerbations by day 21 of treatment Design: randomised, double‐blind, double‐dummy, placebo‐controlled trial Total study duration: not yet published Number of study centres and locations: multi‐centre; Brisbane, Darwin, Melbourne, Perth, and Sydney in Australia, and Auckland in New Zealand Study setting: home Methods of recruitment: chest clinic Analysis by intent‐to‐treat: planned Power calculation: 170 children (85 per arm), providing 90% power (α = 0.05, 1‐sided) with 20% non‐inferiority margin to detect 80% resolution rate by day 21 Trial registration: ANZCTR; ACTRN12612000010897 Conflict of interest: Study authors declare that they have no financial competing interests related to this study. Funders: The study is funded by a 3‐year Australian NHMRC project grant (number 1019834) and is supported by a NHMRC Centre for Research Excellence in Lung Health of Aboriginal and Torres Strait Islander Children grant (number 1040830). A Chang (grant number 545216) is supported by NHMRC practitioner fellowship. KAF O'Grady is supported by funding from the Children's Health Foundation Queensland, Queensland Government Smart Futures fellowship (number 51008) and NHMRC Career Development Fellowship (number 1045157). Ethical approval: Human Research Ethics Committees of all participating institutions (Brisbane: Children's Health Queensland Hospital and Health Service (Royal Children's Hospital) and University of Queensland; Darwin: Department of Health and Families and Menzies School of Health Research; Melbourne: Royal Children's Hospital; Perth: Princess Margaret Hospital; Sydney: Sydney Children's Hospital Network Human Research ethics committee; and Auckland: Northern Ethics Committee, Ministry of Health and Starship Children's Health local ethics committee) |
| Participants |
Inclusion criteria: younger than 18 years; bronchiectasis, as defined by HRCT scan within the previous 5 years OR followed by a respiratory physician for treatment of bronchiectasis if diagnosed earlier; 2 or more respiratory exacerbations of bronchiectasis symptoms in the 18 months before study enrolment Exclusion criteria: cystic fibrosis (sweat chloride > 35 mmol/L or gene mutation); liver dysfunction; severe (hypoxia, dyspnoea, or hospitalisation required) or recent exacerbation (in the 4 weeks before study enrolment; known hypersensitivity to macrolides or penicillins; taking regular maintenance antibiotics of the same class as the investigational antibiotics (macrolides and penicillins); taking macrolides or penicillins within 3 weeks of study enrolment; current or recent (within 4 months before study enrolment) identification of P aeruginosa organism in the airways; current treatment for an oncology condition of any kind |
| Interventions |
Arm 1: daily oral azithromycin 5 mg/kg for 21 days Arm 2: twice‐daily oral amoxicillin‐clavulanic acid 22.5 mg/kg for 21 days These treatments are administered only for the first respiratory exacerbation after enrolment. |
| Outcomes | Detectable difference in PC‐QOL (Parent Cough‐specific Quality of Life) (minimum important difference between groups = 0.9) Detectable difference in serum laboratory assays: CRP, serum amyloid a, IL‐6, IL‐10, IP‐10 (this outcome is assessed only when possible, i.e. not in all participants) Duration of exacerbation symptoms (symptoms of exacerbation are considered increased or changed quality of cough, increased sputum production, or change in patient's normal sputum colour or purulence) Presence and prevalence of viral and bacterial respiratory pathogens and antibiotic (penicillin and macrolide) resistance of pathogens on nasal swabs and sputum samples Requirement for hospitalisation for respiratory exacerbations |
| Starting date | 1/10/2012 |
| Contact information | http://www.anzctr.org.au/ACTRN12612000010897.aspx |
| Notes | Status recorded as "recruitment completed"; details up‐to‐date on 12 June 2017 |
CRP: C‐reactive protein.
IL: interleukin.
IP: IFN‐γ‐induced protein .
NHMRC: Natgional Health and Medical Research Council.
P aeruginosa: Pseudomonas aeruginosa.
PC‐QOL: Patient Cough‐specific Quality of Life.