Nebulised Hypertonic Saline to Decrease Respiratory Exacerbations in Neuromuscular Disease or Neurodisability
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: saline.
- Who it may be relevant to
- Registry conditions: Neuromuscular Diseases, Neurodevelopmental Disorders, Cerebral Palsy. Basic parameters: from 5 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Effectiveness of Nebulised Hypertonic Saline to Decrease Respiratory Exacerbations in People With Neuromuscular Disease or Neurodisability: a Phase 2 Open Label Pilot Study
Overview
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.
Interventions
- Device saline
nebulised
Primary outcome measures
- Course of antibiotics for respiratory infections [Time frame: from baseline to week 52]
Secondary outcome measures (12)
- Lung clearance index [Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.]
- Forced oscillation technique [Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.]
- Forced oscillation technique [Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.]
- Lung ultrasound [Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.]
- Electrical Impedance Tomography [Time frame: at baseline before and within 2 hours after drug response assessment, and at week 52.]
- Airway inflammation [Time frame: baseline and at week 52]
- Airway inflammation [Time frame: baseline and at week 52]
- Airway inflammation [Time frame: baseline and at week 52]
- Airway inflammation [Time frame: baseline and at week 52]
- Bacterial diversity [Time frame: baseline and at week 52]
- Bacterial diversity [Time frame: baseline and at week 52]
- Bacterial diversity [Time frame: baseline and at week 52]
Eligibility criteria
Inclusion criteria
- Diagnosis of NMD or neurodisablity by a physician independent of the study, on standard criteria.
- Age 5 years and above, including adults.
- Must be able to tolerate nebulised 6% hypertonic saline.
- Must have a history of at least one respiratory exacerbation requiring antibiotic treatment with or without the need for hospitalisation in the 12 months prior to recruitment.
Exclusion criteria
- Patients with additional diagnosis, for example, CF, but those with aspiration and/or bronchiectasis secondary to respiratory complications of NMD will be included.
- Patients who are already prescribed daily HS in any concentration (i.e, 3%, 5%, 6%, 7%) will be excluded, but those who are on daily NS or have HS prescribed as part of their escalation plan (i.e., PRN) will be included.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Prevention
Study locations
United Kingdom · 2 centers
- Royal Brompton Hospital — London
- Nottingham University Hospitals — Nottingham
Identifiers
NCT: NCT06134401 · 23IC8597