REPEAT: Reducing Exacerbations in people with primary ciliary dyskinesia (PCD) using Erdosteine and Azithromycin Therapy

Phase 3

Completed

• Conditions: primary ciliary dyskinesia; Respiratory - Other respiratory disorders / diseases; Human Genetics and Inherited Disorders - Other human genetics and inherited disorders

• Registration Number: ACTRN12619000564156
• Lead Sponsor: Menzies School of Health Research

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2019-04-10
• Study Completion: 2024-07-11
• Last Update Posted: 5 August 2024

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 117

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Respiratory exacerbation rate. An acute respiratory episode is defined by an acute respiratory event that: (a) is treated with antibiotics and (b) an increase in sputum volume or purulence, for 3 or more days of change in cough (at least 20% increase in cough score or type [dry to wet/productive]) or physician confirmed acute change in respiratory rate, work of breathing or chest signs.<br>These will be collected by participant reports and medical records.[ 12 months]