Effect of prophylactic azithromycin on chest infections in children with neurological and neuromuscular impairment

Phase 3

Recruiting

Conditions: eurological impairment
Lower respiratory tract infection
Neurological impairment
Respiratory - Other respiratory disorders / diseases
Neurological - Other neurological disorders

Registration Number: ACTRN12621001486819

Lead Sponsor: Menzies School of Health Research

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 94

Inclusion Criteria

1. Children and young people who are aged between 0.5-3 years (inclusive) at randomisation
2. Written informed consent from legal guardian
3. Diagnosed with non-progressive neurological impairment (includes stable neuromuscular abnormalities), AND
4. One or more of the following:
a) Received at least 2 courses of oral antibiotics for LRTI in 26 weeks prior to eligibility
b) Have been hospitalised with a LRTI within 52 weeks prior to eligibility and completed
c) Prescribed prophylactic antibiotics for LRTIs and undergone a 4 week ‘washout’ period'.

Exclusion Criteria

1. Progressive neuromuscular disorders such as Duchenne muscular dystrophy etc., or neurological disorders in which progressive deterioration in neurological condition are known to occur (e.g. Rett syndrome, some neurometabolic syndromes)
2. Pre-existing non-neurological conditions that impact on respiratory function such as cystic fibrosis (CF), immunodeficiency etc. Children with neurological impairment known to have bronchiectasis will not be excluded.
3. Known contra-indication to using (e.g. prolonged QT syndrome) or hypersensitivity to azithromycin, erythromycin, any macrolide or ketolide antibiotic or to any of the excipients contained in the study drug
4. Use of macrolide antibiotics within 90 days prior to eligibility
5. Known significant hepatic disease (hepatic impairment per Child-Pugh classification C)
6. Treatment with ergot derivatives (dihydroergocristine, dihydroergotamine, dihydroergotoxine, nicergoline or a combination of dihydroergocryptine with caffeine)
OR
7. Recruited to another investigation medical product (IMP) trial and continuing to administer the IMP.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Rate of (Lower Respiratory Tract Infection) LRTIs measured in child-months assessed by both parent self-report and medical reports.[ 6 months from commencement of trial medications given]

Secondary Outcome Measures

Name	Time	Method
Proportion of children with LRTI over the 26-weeks intervention period, assessed by both parent self-report and medical reports.[ 6 months from time trial medications commenced];Adverse events assessed by both parent self-report (on diaries) and medical reports. These include but are not restricted to mild diarrhoea, abdominal pains, nausea or vomiting, headache and dizziness.[ During the 6 months of the intervention period];Quality-adjusted life years (QALY) assessment (composite outcome).<br><br>CHU9D and EQ-5D-Y are used to evaluate the outcome measure for QALY. However, as not all the questions are applicable for the young children i.e. only applicable questions (for age) will be used.[ Baseline and at 6 months from commencement of trial medications];Change in health related QoL of parent/carer using Parent QoL assessment (Warwick-Edinburgh)[ Baseline and at 6 months from commencement of trial medications];Respiratory symptom questionnaire[ Baseline and at 6 months from commencement of trial medications]