None listed
Conditions
Brief summary
Neurological Impairment (NI) in children is often caused by conditions such as cerebral palsy. Many children with NI are prone to chest infections which can lead to long stays in hospital, additional impairment and even premature death. Despite the suffering caused to children and their families by these infections and the high cost to health services, there is very little information on how best to prevent them. Some doctors prescribe long-term antibiotics but we don't really know whether this treatment makes any difference to the numbers of chest infections children suffer from, or whether these antibiotics can cause long term harm. The trial is looking to recruit for 500 children and young people aged 3-17 years, with NI who are at risk of chest infections, along with their parents / primary care giver to take part. Children included in the trial will be given either azithromycin or a placebo for 12 months to compare the difference. The trial is taking place in the UK and Australia and each participant will be involved for a maximum of 18 months. The aim of the trial is to find out whether 12-month's treatment with the antibiotic azithromycin reduces how often children with NI have to stay in hospital with chest infections.
Interventions
Arm 1: Azithromycin
The dosing regimen is based on body weight (10mg/kg rounded) and will be administered as an oral suspension 3 times weekly (Mon/Wed/Fri) for 52 weeks.
Adherence will be monitored at 13, 26, 39 and 52 weeks using an IMP treatment diary and withdrawals from study treatment will also be monitored.
Sponsors
University of Liverpool
Study design
Allocation
Randomised controlled trial
Intervention model
Parallel
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
3 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
1. Children and young people who are aged between 3-17 years (inclusive) at randomisation 2. Written informed consent from participant (or appropriate person if incapacitated / minor) 3. Participant (or appropriate person if incapacitated / underage) and caregiver have a good understanding of the English language 4. Diagnosed with non-progressive, non-neuromuscular NI 5. Persistent respiratory symptoms* 6. One or more of the following: a) Received at least 2 courses of oral antibiotics for LRTI in 52 weeks prior to eligibility b) Have been hospitalised with a LRTI within 52 weeks prior to eligibility and completed 13 week ‘washout’ period (where applicable)** c) Prescribed prophylactic antibiotics for LRTIs and undergone a 13 week ‘washout’ period**. * Defined by: LRSQ-Neuro score of equal to or higher than 95%CI for age: Age (in years) LRSQ-Neuro total score greater than or equal to 3 and less than 6 greater than or equal to 11 greater than or equal to 6 and less than 11 greater than or equal to 5 greater than or equal to 11 and less than or equal to 17 greater than or equal to4 **Must have undergone a 13 week ‘washout’ period where administered IV antibiotics during hospitalisation or have been previously prescribed and administered prophylactic antibiotics.
Exclusion criteria
1. Any neuromuscular disorders including SMA, 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. Note: Children with NI 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) 7. Child/young person already taking prophylactic antibiotics for non-respiratory causes (e.g. UTIs). 8. Previously randomised in PARROT 9. Recruited to another IMP trial and continuing to administer the IMP.
Outcome results
None listed