Bronchiectasis, Bronchopulmonary Dysplasia (BPD), Chronic Atelectasis, Cystic Lung Disease, Tracheomalacia, Vascular Ring
Conditions
Brief summary
Children with lung and airway malformations or early structural lung damage face significant challenges, often leading to recurrent respiratory infections, hospitalizations, and decreased quality of life. Despite various interventions, effective strategies are urgently needed. The link between these conditions and persistent bacterial bronchitis remains unclear, possibly due to compromised airways and reduced mucociliary clearance. Although antibiotics can alleviate symptoms, relapse is common. Experts often prescribe prophylactic azithromycin, despite limited evidence of its benefits. Azithromycin shows promise due to its anti-inflammatory and immunomodulatory effects but lacks thorough evaluation in this population. To address this gap, we propose a double-blind, randomized controlled trial to assess azithromycin's effectiveness and safety in preventing respiratory infections in children with these conditions. This research aims to inform clinical practice and improve the health of affected children and their families.
Interventions
DRUGAzithromycin 40 MG/ML
Maintenance Antibiotic treatment
OTHERPlacebo
Placebo
Sponsors
Rigshospitalet, Denmark
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
The study comprises a multicenter, parallel-group, double-blind, randomized placebo-controlled clinical trial (RCT) with a single intervention.
Eligibility
Sex/Gender
ALL
Age
0 Months to 72 Months
Healthy volunteers
No
Inclusion criteria
1. CT and bronchoscopy verified and structural lung damage or congenital lung and airway malformations 2. Outpatient affiliation with one of the three highly specialized pediatric pulmonology centers. 3. At risk of or already documented respiratory infections requiring antibiotics. 4. Age between 0-72 months at inclusion.
Exclusion criteria
1. Asthmatic challenges: Patients without any of the conditions mentioned in table 1 repeatedly experiencing asthmatic problems are not eligible for this study. 2. Cystic fibrosis (CF) or primary ciliary dyskinesia (PCD): Patients with a CF or PCD diagnosis will be excluded. 3. Impaired liver function: Children with an alanine transaminase (ALAT) twice or more the upper limits of normal will be excluded. 4. Impaired kidney function: Children with a serum creatinine higher than the upper limit of normal for age will be excluded. 5. Neurological or psychiatric disorders 6. Prolonged QT interval: Patients with either congenital or acquired prolonged QT interval will be excluded. 7. Heart disease: Patients with clinically relevant bradycardia, cardiac arrhythmia or severe heart failure are not eligible for this study. 8. Allergy to macrolide antibiotics: documented allergy to macrolide antibiotics (extremely rare) will result in exclusion from the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of exacerbations | 6 months | Number of exacerbations where systemic antibiotics is needed within the study period |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Severity of exacerbations | 6 months | Duration of exacerbations, hospitalizations and durations of hospitalizations |
Other
| Measure | Time frame | Description |
|---|---|---|
| Lung function measures | 1 year | Impulse oscillometry (IOS) measurement pre- and post drug administrations |
| Quality of Life | 1 year | Questionaire on a Likert scale 0-5. Using PedsQL(TM). |
Outcome results
None listed