Nebulised Hypertonic Saline to Decrease Respiratory Exacerbations in Neuromuscular Disease or Neurodisability

NCT06134401 | Not Yet Recruiting

• 1 other identifier: 23IC8597
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 2

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.

Conditions

Neuromuscular Diseases; Neurodevelopmental Disorders; Cerebral Palsy

Keywords

hypertonic saline; saline; nebulised

Outcome Measures

Primary Outcomes (1)

• 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.

  from baseline to week 52

Secondary Outcomes (18)

• Lung clearance index

  at baseline before and within 2 hours after drug response assessment, and at week 52.

• Forced oscillation technique

  at baseline before and within 2 hours after drug response assessment, and at week 52.

• Forced oscillation technique

  at baseline before and within 2 hours after drug response assessment, and at week 52.

• Lung ultrasound

  at baseline before and within 2 hours after drug response assessment, and at week 52.

• Electrical Impedance Tomography

  at baseline before and within 2 hours after drug response assessment, and at week 52.

Other Outcomes (16)

• Recruitment rate

  1 year

• Consent rate

  1 year

• Retention rate

  1 year

Study Arms (2)

Treatment

EXPERIMENTAL

Nebulised 6% Hypertonic saline

Device: saline

Control

PLACEBO COMPARATOR

Nebulised 0.9% normal saline

Device: saline

Interventions

saline DEVICE

nebulised

Control; Treatment

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Imperial College London: lead
• Nottingham University Hospitals NHS Trust: collaborator
• Royal Brompton & Harefield NHS Foundation Trust: collaborator
• Pari Pharma GmbH: collaborator

Study Sites (2)

Royal Brompton Hospital

London, SW3 6NP, United Kingdom

Location

Nottingham University Hospitals

Nottingham, NG7 2UH, United Kingdom

Location

MeSH Terms

Conditions

Neuromuscular Diseases; Neurodevelopmental Disorders; Cerebral Palsy

Condition Hierarchy (Ancestors)

Nervous System Diseases; Mental Disorders; Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases

Study Officials

• Andrew Bush, Professor

  Imperial College London

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Natalia G Galaz Souza

CONTACT

0787131892; natalia.galaz-souza16@imperial.ac.uk

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 3, 2023
• First Posted: November 18, 2023
• Study Start: June 1, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2027
• Last Updated: October 15, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data will become available after all study activities have finished. It will be available for 10 years.
• Access Criteria: Data ownership rights will lie with the institution

Locations

GB (2)