Improving outcomes of children and adults with primary ciliary dyskinesia: a multi-centre, double-blind, double-dummy, 2x2 partial factorial, randomised controlled trial using azithromycin and erdosteine

Menzies School of Health Research0 个研究点目标入组 117 人开始时间: 2019年4月10日最近更新: 2024年8月5日

适应症

试验速览

暂无简介。

•1.Children or adults (aged 2-.65 years)
•2.Known PCD (defined by genetics or through electron microscopy) or probable PCD defined as (a) chronic wet/productive cough and a PICADAR score of at least 5 with nasal nitric oxide measurement (nNO) less than 77 nL/min [or equivalent cut-offs] on 2 occasions (when nNO levels can be measured i.e. aged over 5 years) or (b) clinician assessment based on medical history
•3.At least two exacerbations in the last 18 months or one hospitalisation for respiratory exacerbation in the last 18 months; AND
•4. Plan to remain at one of the study sites for at least 15 months

•1.Cystic fibrosis
•2.On regular azithromycin (within the last 8 weeks) but if a participant is too unstable off azithromycin (defined by the participant’s primary specialist), the participant may be enrolled and will just be allocated to the erdosteine/placebo component (1:1 allocation)
•3.Past (last 6 months) or current infection with non-tuberculous mycobacteria
•4.Contraindication for macrolide or erdosteine use (e.g. liver dysfunction, hypersensitivity, renal failure, deficiency of the cystathionine-synthetase enzyme, phenylketonuria, active peptic ulcer)
•5.Pregnant, pregnancy planned (in next 12 months) or nursing mothers
•6. Abnormal ECG (QTc over 460 msec) or history of cardiac arrhythmia
•7. Previously randomised
•8. Hospitalised in the last 4 weeks for respiratory instability

发起方