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N/A N=24

Nebulised Hypertonic Saline in Children and Young People With Neuromuscular Disease and Cerebral Palsy

Neuromuscular Diseases

Enrolled (actual)
24
Serious AEs
Results posted
Sep 2020
Primary outcome: Primary: Change in Antibiotic Courses — 4; 1; 2.50; 1.00 Courses of antibiotics — p=<0.001

Study Design & Population

Study type
Observational
Phase
N/A
Interventions
Nebulised hypertonic saline (Drug)
Age
Pediatric, Adult · 1+ yrs
Sex
All
Sponsor
Imperial College London
Primary completion
Aug 2019

Outcome Measures

OutcomeResultp-value
PRIMARY
Change in Antibiotic Courses
4; 1; 2.50; 1.00; 1.00; 0.00 <0.001 sig
PRIMARY
Number of Hospitalsations Due to Respiratory Exacerbations
1; 0 0.001 sig
SECONDARY
Participant's Perception of Treatment
11; 0; 0
SECONDARY
Parent's or Legal Guardian's Perception of Treatment
15; 9; 0; 0; 0
SECONDARY
Score on the Ease of Airway Clearance Pictorial Analogue Scale From Children and Young Adults as Participants
6.45; 2 0.003 sig
SECONDARY
Score on the Ease of Airway Clearance From Parents or Legal Guardians
4.21; 2.13 <0.001 sig
SECONDARY
Apnea Index (AI)
4.87; 0.40
SECONDARY
AHI
8.53; 1.38
SECONDARY
%SpO2
96.55; 96.53 0.97
SECONDARY
Nocturnal ODI
4.26; 3.32 0.09
SECONDARY
TcPCO2
5.77; 6.00 0.65
SECONDARY
FEV1/FVC %Predicted Rate of Decline
97.37; 98.89
SECONDARY
FEV1% Predicted Rate of Decline
-8.03; 4.00
SECONDARY
FVC% Predicted Rate of Decline
-7.44; 7.00
SECONDARY
Peak Expiratory Flow (PEF)
43.5; 47

Summary

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.

Eligibility Criteria

Inclusion Criteria

  • Children with Neuromuscular disease or Cerebral Palsy who have been on treatment with nebulised Hypertonic Saline for at least 12 months.

Exclusion Criteria

  • Children also diagnosed with cystic fibrosis.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03623698). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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