An interventional study of saline in Neuromuscular Diseases, Neurodevelopmental Disorders and Cerebral Palsy, sponsored by Imperial College London. Not yet recruiting at 2 sites in United Kingdom. Open to participants aged 5 Years and older. Per ClinicalTrials.gov, last updated 2024-10-15.
Sponsored by Imperial College London · Not applicable, Interventional, and Prevention
Research Aim: This study investigates whether a 12-month treatment with hypertonic saline (salty water) can reduce antibiotic use in individuals with neuromuscular disease or cerebral palsy who frequently experience chest infections due to difficulty clearing mucus from their airways.
Methodology: Participants will be randomly assigned to receive nebulised hypertonic saline (7% salt in water) or normal saline (0.9% salt in water). The study is open-label as both participants and researchers are aware of the treatment, necessary due to the differing tastes of the solutions. Two centers, Royal Brompton Hospital in London and Queens Medical Centre in Nottingham, will conduct the research.
Before starting the treatment, participants will undergo various assessments, including questionnaires to measure quality of life and treatment satisfaction, sputum/throat swab collection, lung clearance index, forced oscillation technique, electrical impedance tomography, and lung ultrasound. Once these assessments are completed, participants will take the assigned treatment at home, administered twice daily for 12 months, with monthly follow-ups regarding difficulties and chest infections. After 12 months, the treatment will cease, and participants will repeat the assessments.
Significance: This research will provide valuable insights into the efficacy of nebulised hypertonic saline for individuals with neuromuscular disease or cerebral palsy, potentially aiding both patients and doctors in making informed treatment decisions.
Dissemination: The study's findings will be shared through publication in scientific journals and presentation at conferences.
1,853 studies on the registry are indexed under Cerebral Palsy; 435 are open to participants now.
This study's planned enrollment of 40 is above the median of 33 across 1,368 interventional studies indexed under Cerebral Palsy.
Browse Cerebral Palsy studies →Imperial College London is the lead sponsor of 824 studies on the registry; 178 are open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 6 (67%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Nebulised 6% Hypertonic saline
Device: saline
Nebulised 0.9% normal saline
Device: saline
nebulised
Course of antibiotics for respiratory infections
Full courses of antibiotics as prescribed for respiratory infections, both oral and intravenous, (excluding prophylactic antibiotic prescriptions). 1 course of antibiotic would be the full treatment for one event of respiratory infection, irrespective of the number of days that the course was prescribed for.
Time frame: from baseline to week 52
Lung clearance index
measured by multiple breath washout
Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.
Forced oscillation technique
Respiratory resistance (Rrs)
Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.
Forced oscillation technique
Respiratory reactance (Xrs).
Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.
Lung ultrasound
Global Lung ultrasound score. The global lung ultrasound score (LUS) quantifies lung aeration by translating lung ultrasound patterns into a numerical score across 12 lung regions (six areas on each side of the chest: two ventral regions, two lateral regions, and two posterolateral regions) and summing the results. The aeration pattern observed in each region is scored from 0 to 3 as follows: 0 = A pattern with ≤2 B lines; 1 = \>2 separated B lines that cover ≤50% of the pleural line; 2 = B lines that cover \>50% of the pleural line; or 3 = lung consolidation. In theory, the global LUS score can range from 0 (normal aeration in all regions) to 36 (severe abnormal aeration in all regions).
Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.
Electrical Impedance Tomography
Electrical impedance tomography (EIT)-based global inhomogeneity index (quantification of homogeneity of the tidal volume distribution). The image matrix in EIT consists of 32 × 32 pixels. Global inhomogeneity (GI) is calculated as the sum of the absolute differences between the median value of tidal variation and every single pixel value, divided by the sum of all impedance values, to normalise the calculated values.The smaller the GI, the more homogeneous the tidal volume is distributed within the ventilated area. A GI of zero represents a perfectly homogeneous distribution of ventilation.
Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.
Airway inflammation
Levels of IL-8 in sputum or throat swab.
Time frame: baseline and at week 52
Airway inflammation
Levels of IL-6 in sputum or throat swab.
Time frame: baseline and at week 52
Airway inflammation
Levels of TNF-a in sputum or throat swab.
Time frame: baseline and at week 52
Airway inflammation
Levels of IL-1b in sputum or throat swab.
Time frame: baseline and at week 52
Bacterial diversity
Operational taxonomic unit (OTU) Richness, defined as count of different species/OTUs.
Time frame: baseline and at week 52
Bacterial diversity
Pielou's eveness index. Pielou's evenness is an index that measures diversity along with species richness.
Time frame: baseline and at week 52
Bacterial diversity
Shannon diversity index. The index takes into account the number of species living in a habitat (richness) and their relative abundance (evenness).
Time frame: baseline and at week 52
Bacterial diversity
Bray-Curtis dissimilarity index. Examines the abundances of microbes that are shared between two samples, and the number of microbes found in each.
Time frame: baseline and at week 52
Ease of airway clearance
0-10 Visual analogue scale, where 0 is most easy, and 10 is most difficult.
Time frame: Once monthly for 52 weeks.
Health-related quality of life
Pediatric Quality of Life Inventory (PedsQL)™. 0-100 scale, where higher scores indicate better HRQOL (Health-Related Quality of Life).
Time frame: At baseline and at week 51.
Patient and main carer treatment satisfaction
Treatment Satisfaction Questionnaire for Medication (TSQM Version 1.4). Scores range from 0 to 100, with higher scores indicating higher satisfaction.
Time frame: weeks 12, 26, 39 and 51
Family impact
PedsQL™ Family Impact Module. The scale has five Likert response options, 'never', 'almost never', 'sometimes', 'often' and 'almost always' (corresponding to scores of 100, 75, 50, 25 and 0). Regarding the interpretation of the scale, higher scores indicate better functioning (less negative impact).
Time frame: Baseline and at week 51.
Health economics
Quality-adjusted life years
Time frame: baseline, week 26 and week 51.
Recruitment rate
Number of participants recruited per centre per month.
Time frame: 1 year
Consent rate
Percentage of eligible participants who consented and were randomised.
Time frame: 1 year
Retention rate
Percentage of randomised participants retained with valid primary outcome data.
Time frame: 1 year
Adherence
Mean percentage of adherence calculated from returned ampoules count
Time frame: 52 weeks
Adherence
Pick-up rate as the percentage of picked up prescriptions from total prescribed doses
Time frame: 52 weeks
Adherence
The Medication Adherence Report Scale (MARS) Score, where each of the 5 items are summed to give a scale score ranging from 5 to 25, where higher scores indicate higher levels of reported adherence.
Time frame: 52 weeks
Compliance with monthly follow-up
Percentage of compliance with completion of monthly questionnaires.
Time frame: 52 weeks
Success rates of outcome measures
Percentage of participants who provided a sputum sample or throat swab
Time frame: 1 year
Success rates of outcome measures
Percentage of participants who completed acceptable measurements of Lung clearance index
Time frame: 1 year
Success rates of outcome measures
Percentage of participants who completed acceptable measurements of Forced oscillation technique
Time frame: 1 year
Success rates of outcome measures
Percentage of participants who completed acceptable measurements of Lung ulstrasound
Time frame: 1 year
Success rates of outcome measures
Percentage of participants who completed acceptable measurements of Electrical impedance tomography
Time frame: 1 year
Time required to complete outcome measures
Time in minutes to complete acceptable measurements of Lung clearance index
Time frame: 2 hours
Time required to complete outcome measures
Time in minutes to complete acceptable measurements of forced oscillation technique
Time frame: 2 hours
Inter-rater reliability of Lung ultrasound analysis
Degree of agreement among independent observers using Cohen Kappa. Cohen suggested the Kappa result be interpreted as follows: values ≤ 0 as indicating no agreement and 0.01-0.20 as none to slight, 0.21-0.40 as fair, 0.41- 0.60 as moderate, 0.61-0.80 as substantial, and 0.81-1.00 as almost perfect agreement.
Time frame: 1 year
Inter-rater reliability of Electrical impedance tomography analysis
Degree of agreement among independent observers using Cohen Kappa. Cohen suggested the Kappa result be interpreted as follows: values ≤ 0 as indicating no agreement and 0.01-0.20 as none to slight, 0.21-0.40 as fair, 0.41- 0.60 as moderate, 0.61-0.80 as substantial, and 0.81-1.00 as almost perfect agreement.
Time frame: 1 year
Plan to share: Yes — Data ownership rights will lie with the institution
Supporting information: Study protocol, Sap, Icf, Csr
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Imperial College London