Clinical trial NCT06134401: Nebulised Hypertonic Saline to Decrease Respiratory Exacerbations in Neuromuscular Disease or Neurodisability
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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Neuromuscular Diseases
Neurodevelopmental Disorders
Cerebral Palsy
Keywords: hypertonic saline
saline
nebulised
Lead sponsor: Imperial College London
Sponsor class: OTHER
Collaborators: name: Nottingham University Hospitals NHS Trust
name: Royal Brompton & Harefield NHS Foundation Trust
name: Pari Pharma GmbH
First posted: 2023-11-18
Enrollment: 40
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

Brief summary: 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.


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.

Primary outcomes:
measure: Course of antibiotics for respiratory infections
timeFrame: from baseline to week 52
Secondary outcomes:
measure: Lung clearance index
timeFrame: at baseline before and within 2 hours after drug response assessment, and at week 52.
measure: Forced oscillation technique
timeFrame: at baseline before and within 2 hours after drug response assessment, and at week 52.
measure: Forced oscillation technique
timeFrame: at baseline before and within 2 hours after drug response assessment, and at week 52.
measure: Lung ultrasound
timeFrame: at baseline before and within 2 hours after drug response assessment, and at week 52.
measure: Electrical Impedance Tomography
timeFrame: at baseline before and within 2 hours after drug response assessment, and at week 52.
measure: Airway inflammation
timeFrame: baseline and at week 52
measure: Airway inflammation
timeFrame: baseline and at week 52
measure: Airway inflammation
timeFrame: baseline and at week 52
measure: Airway inflammation
timeFrame: baseline and at week 52
measure: Bacterial diversity
timeFrame: baseline and at week 52
measure: Bacterial diversity
timeFrame: baseline and at week 52
measure: Bacterial diversity
timeFrame: baseline and at week 52
measure: Bacterial diversity
timeFrame: baseline and at week 52
measure: Ease of airway clearance
timeFrame: Once monthly for 52 weeks.
measure: Health-related quality of life
timeFrame: At baseline and at week 51.
measure: Patient and main carer treatment satisfaction
timeFrame: weeks 12, 26, 39 and 51
measure: Family impact
timeFrame: Baseline and at week 51.
measure: Health economics
timeFrame: baseline, week 26 and week 51.
Other outcomes:
measure: Recruitment rate
timeFrame: 1 year
measure: Consent rate
timeFrame: 1 year
measure: Retention rate
timeFrame: 1 year
measure: Adherence
timeFrame: 52 weeks
measure: Adherence
timeFrame: 52 weeks
measure: Adherence
timeFrame: 52 weeks
measure: Compliance with monthly follow-up
timeFrame: 52 weeks
measure: Success rates of outcome measures
timeFrame: 1 year
measure: Success rates of outcome measures
timeFrame: 1 year
measure: Success rates of outcome measures
timeFrame: 1 year
measure: Success rates of outcome measures
timeFrame: 1 year
measure: Success rates of outcome measures
timeFrame: 1 year
measure: Time required to complete outcome measures
timeFrame: 2 hours
measure: Time required to complete outcome measures
timeFrame: 2 hours
measure: Inter-rater reliability of Lung ultrasound analysis
timeFrame: 1 year
measure: Inter-rater reliability of Electrical impedance tomography analysis
timeFrame: 1 year
Locations:
city: London
country: United Kingdom
facility: Royal Brompton Hospital
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospitals
Study officials:
affiliation: Imperial College London
name: Andrew Bush, Professor
role: PRINCIPAL_INVESTIGATOR
References: