Adults 1 to 18, any sex, with Neuromuscular Diseases. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change in Antibiotic CoursesPrimary· Change from baseline (before treatment) and 12 months after treatment
Treatments due to respiratory exacerbations
Total courses of antibiotics
Group
Value
95% CI
Before Treatment
4
2 – 6
After Treatment
1
0 – 2
Courses of Oral antibiotics
Group
Value
95% CI
Before Treatment
2.50
1.00 – 6.00
After Treatment
1.00
0.00 – 2.00
Courses of intravenous antibiotics
Group
Value
95% CI
Before Treatment
1.00
0.00 – 1.00
After Treatment
0.00
0.00 – 0.00
Number of Hospitalsations Due to Respiratory ExacerbationsPrimary· Change from baseline (before treatment) and 12 months after treatment
Number of respiratory exacerbations that required not planned hospitalisation
Group
Value
95% CI
Before Treatment
1
0 – 2
After Treatment
0
0 – 0
Participant's Perception of TreatmentSecondary· At 12 months after starting treatment with hypertonic saline
Questionnaire: "Hypertonic saline treatment questionnaire". Perception of overall usefulness of nebulised hypertonic saline: "Useful", "Not useful", "I don't know".
Useful
Group
Value
95% CI
Children and Young People With Neuromuscular Disease
11
Not useful
Group
Value
95% CI
Children and Young People With Neuromuscular Disease
0
I don't know
Group
Value
95% CI
Children and Young People With Neuromuscular Disease
0
Parent's or Legal Guardian's Perception of TreatmentSecondary· At 12 months after starting treatment with hypertonic saline
"Hypertonic saline treatment questionnaire for legal guardian". Measures the perception of overall usefulness of nebulised hypertonic saline through a likert scale: Very useful, useful, neither useful or not useful, not useful, not at all useful.
Very useful
Group
Value
95% CI
Parent's or Legal Guardians
15
useful
Group
Value
95% CI
Parent's or Legal Guardians
9
neither useful or not useful
Group
Value
95% CI
Parent's or Legal Guardians
0
not useful
Group
Value
95% CI
Parent's or Legal Guardians
0
not at all useful
Group
Value
95% CI
Parent's or Legal Guardians
0
Score on the Ease of Airway Clearance Pictorial Analogue Scale From Children and Young Adults as ParticipantsSecondary· Change from baseline (before treatment) and 12 months after treatment
Pictorial visual scale "Facial Rating of perceived exertion Scale". Measures ease of airway clearance. Values range starting in 0 (Extremely easy) to 10 (Extremely hard), including 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10. Numbers are also associated with facial expressions.
Group
Value
95% CI
Before Treatment
6.45
4 – 9
After Treatment
2
0 – 4
Score on the Ease of Airway Clearance From Parents or Legal GuardiansSecondary· Change from baseline (before treatment) and 12 months after treatment
Measures ease of airway clearance through a 1-5 likert scale: 1) Very easy, 2) Easy, 3) Neither easy nor difficult, 4) Not easy, 5) Not at all easy.
Group
Value
95% CI
Before Treatment
4.21
3 – 5
After Treatment
2.13
1 – 3
Apnea Index (AI)Secondary· Change from baseline (before treatment) and 12 months after treatment
The number of apneas recorded during the study per hour of sleep
Group
Value
95% CI
Before Treatment
4.87
± 1.02
After Treatment
0.40
± 0.4
AHISecondary· Change from baseline (before treatment) and 12 months after treatment
Nocturnal Apnoea Hipopnea index: total number of apnea events plus hypopnea events divided by the total number of minutes of actual sleep time and then multiplied by 60.
Group
Value
95% CI
Before Treatment
8.53
± 1.70
After Treatment
1.38
± 0.99
%SpO2Secondary· Change from baseline (before treatment) and 12 months after treatment
Nocturnal oxygen saturation
Group
Value
95% CI
Before Treatment
96.55
± 1.92
After Treatment
96.53
± 1.83
Nocturnal ODISecondary· Change from baseline (before treatment) and 12 months after treatment
Oxygen desaturation index: Number of desaturations per hour of sleep
Group
Value
95% CI
Before Treatment
4.26
± 3.42
After Treatment
3.32
± 3.33
TcPCO2Secondary· Change from baseline (before treatment) and 12 months after treatment
Nocturnal Transcutaneous Carbon Dioxide in kPa
Group
Value
95% CI
Before Treatment
5.77
± 0.95
After Treatment
6.00
± 1.04
FEV1/FVC %Predicted Rate of DeclineSecondary· Change from baseline (before treatment) and 12 months after treatment
Rate of decline per year of Tiffenau index
Group
Value
95% CI
Before Treatment
97.37
84.50 – 110.50
After Treatment
98.89
92.00 – 109.00
Sponsor's own description
Pneumonia, respiratory exacerbations, and chronic pulmonary infection are important causes of emergency admissions, hospitalisations and death in children with Neuromuscular disorders and Cerebral Palsy. Hence, there is a need for research on how to therapeutically aid airway clearance and decrease respiratory exacerbations. Studies have shown that nebulised Hypertonic Saline is well tolerated, reduces pulmonary exacerbations and improves lung function and Lung Clearance Index in patients with Cystic Fibrosis, and enhances mucociliary clearance in asthmatic patients. Nevertheless, to the investigators' knowledge, there is no available data concerning the use of nebulised Hypertonic Saline in the management of children with Neuromuscular disorders and Cerebral Palsy. This study aims to assess the effectiveness of nebulised Hypertonic Saline to decrease hospitalisations and courses of antibiotics in children with Neuromuscular disorders and Cerebral Palsy.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Imperial College London
Last refreshed: 16 September 2020
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03623698.