Neuromuscular DiseasesSPICE-UPNCT06134401
Nebulised Hypertonic Saline to Decrease Respiratory Exacerbations in Neuromuscular Disease or Neurodisability
A study testing saline in people with Neuromuscular Diseases and Neurodevelopmental Disorders, run by Imperial College London at 2 sites in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Neuromuscular Diseases, Neurodevelopmental Disorders, Cerebral Palsy
- What is being tested?
- saline
- Type of treatment
- Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- Jun 2025to Dec 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United Kingdom2 sites
Royal Brompton Hospital
London, SW3 6NP
Natalia Galaz Souza, +447871331892, natalia.galaz-souza16@imperial.ac.uk
natalia.galaz-souza16@imperial.ac.uk
Nottingham University Hospitals
Nottingham, NG7 2UH
Natalia G Souza, 07871331892, natalia.galaz-souza16@imperial.ac.uk
Natalia Galaz Souza
About this study
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
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.
Read more
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.
- Being tested
- saline
- Keywords
- hypertonic saline, saline, nebulised
- Medical subject headings
- Neuromuscular Diseases, Neurodevelopmental Disorders, Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
salineDevice
nebulised
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Nebulised 6% Hypertonic saline Receives: Device: saline |
| Control | Placebo comparator | Nebulised 0.9% normal saline Receives: Device: saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: from baseline to week 52
Secondary outcomes (18)
Lung clearance index
measured by multiple breath washout
Measured: at baseline before and within 2 hours after drug response assessment, and at week 52.
Forced oscillation technique
Respiratory resistance (Rrs)
Measured: at baseline before and within 2 hours after drug response assessment, and at week 52.
Forced oscillation technique
Respiratory reactance (Xrs).
Measured: at baseline before and within 2 hours after drug response assessment, and at week 52.
Lung ultrasound
Global Lung ultrasound score. The global lung ultrasound score (LUS) quantifies lung aeration by translating lung ultrasound patterns into a numerical score across 12 lung regions (six areas on each side of the chest: two ventral regions, two lateral regions, and two posterolateral regions) and summing the results. The aeration pattern observed in each region is scored from 0 to 3 as follows: 0 = A pattern with ≤2 B lines; 1 = >2 separated B lines that cover ≤50% of the pleural line; 2 = B lines that cover >50% of the pleural line; or 3 = lung consolidation. In theory, the global LUS score can range from 0 (normal aeration in all regions) to 36 (severe abnormal aeration in all regions).
Measured: at baseline before and within 2 hours after drug response assessment, and at week 52.
Electrical Impedance Tomography
Electrical impedance tomography (EIT)-based global inhomogeneity index (quantification of homogeneity of the tidal volume distribution). The image matrix in EIT consists of 32 × 32 pixels. Global inhomogeneity (GI) is calculated as the sum of the absolute differences between the median value of tidal variation and every single pixel value, divided by the sum of all impedance values, to normalise the calculated values.The smaller the GI, the more homogeneous the tidal volume is distributed within the ventilated area. A GI of zero represents a perfectly homogeneous distribution of ventilation.
Measured: at baseline before and within 2 hours after drug response assessment, and at week 52.
Airway inflammation
Levels of IL-8 in sputum or throat swab.
Measured: baseline and at week 52
Airway inflammation
Levels of IL-6 in sputum or throat swab.
Measured: baseline and at week 52
Airway inflammation
Levels of TNF-a in sputum or throat swab.
Measured: baseline and at week 52
Airway inflammation
Levels of IL-1b in sputum or throat swab.
Measured: baseline and at week 52
Bacterial diversity
Operational taxonomic unit (OTU) Richness, defined as count of different species/OTUs.
Measured: baseline and at week 52
Bacterial diversity
Pielou's eveness index. Pielou's evenness is an index that measures diversity along with species richness.
Measured: baseline and at week 52
Bacterial diversity
Shannon diversity index. The index takes into account the number of species living in a habitat (richness) and their relative abundance (evenness).
Measured: baseline and at week 52
Bacterial diversity
Bray-Curtis dissimilarity index. Examines the abundances of microbes that are shared between two samples, and the number of microbes found in each.
Measured: baseline and at week 52
Ease of airway clearance
0-10 Visual analogue scale, where 0 is most easy, and 10 is most difficult.
Measured: Once monthly for 52 weeks.
Health-related quality of life
Pediatric Quality of Life Inventory (PedsQL)™. 0-100 scale, where higher scores indicate better HRQOL (Health-Related Quality of Life).
Measured: At baseline and at week 51.
Patient and main carer treatment satisfaction
Treatment Satisfaction Questionnaire for Medication (TSQM Version 1.4). Scores range from 0 to 100, with higher scores indicating higher satisfaction.
Measured: weeks 12, 26, 39 and 51
Family impact
PedsQL™ Family Impact Module. The scale has five Likert response options, 'never', 'almost never', 'sometimes', 'often' and 'almost always' (corresponding to scores of 100, 75, 50, 25 and 0). Regarding the interpretation of the scale, higher scores indicate better functioning (less negative impact).
Measured: Baseline and at week 51.
Health economics
Quality-adjusted life years
Measured: baseline, week 26 and week 51.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2023
- Study startJun 2025 (estimated)
- Primary completionDec 2026 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseasesStudies of the same condition, open and under way.
- More trials run by Imperial College LondonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Imperial College London
- Sponsor type
- University, hospital or other
- Collaborators
- Nottingham University Hospitals NHS Trust, Royal Brompton & Harefield NHS Foundation Trust, Pari Pharma GmbH
- Responsible party
- Sponsor
- Investigators
- Andrew Bush, Professor, Principal investigator, Imperial College London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06134401
- Sponsor's protocol ID
- 23IC8597
- Acronym
- SPICE-UP
- Status last verified
- Oct 2024
- First submitted
- 3 Nov 2023
- Last updated
- 15 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Data ownership rights will lie with the institution
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06134401
- Acronym / sponsor ID
- SPICE-UP / 23IC8597
- Keywords
- hypertonic saline, saline, nebulised
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Imperial College LondonUniversity, hospital or other
- Collaborators
- Nottingham University Hospitals NHS Trust, Royal Brompton & Harefield NHS Foundation Trust, Pari Pharma GmbH
- Study start
- Jun 2025 (estimated)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 18 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Device: salineNebulised 6% Hypertonic saline |
| Control | Placebo comparator | Device: salineNebulised 0.9% normal saline |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| salinenebulised | Device | Control, Treatment |
Primary outcome
Course of antibiotics for respiratory infections
Time frame: from baseline to week 52
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.
18 secondary outcomes
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
Bacterial diversity
Time frame: baseline and at week 52
Ease of airway clearance
Time frame: Once monthly for 52 weeks.
Health-related quality of life
Time frame: At baseline and at week 51.
Patient and main carer treatment satisfaction
Time frame: weeks 12, 26, 39 and 51
Family impact
Time frame: Baseline and at week 51.
Health economics
Time frame: baseline, week 26 and week 51.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2024.
- Individual participant dataSharing planned: Yes. Data ownership rights will lie with the institution
Related trials for Neuromuscular Diseases
Not yet recruiting
Effects of Adding Vagal Neuromodulation to Manual Therapy in Women With Fibromyalgia
Fibromyalgia (FM) OR Fibromyalgia Syndrome, Manuel Therapy
Active, not recruitingObservational study
A Study of the Safety of Nusinersen (Spinraza®) and Its Effects on Motor Function in Koreans Living With SMA Treated During Their Usual Medical Care
Muscular Atrophy, Spinal
Active, not recruitingObservational study
Possible Sarcopenia, Sarcopenia, and Functional Status in Patients With Primary Sjögren's Syndrome
Primary Sjögren Syndrome, Sarcopenia
RecruitingObservational study
Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others
Charcot Marie Tooth Disease
Recruiting
Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders
Cerebral Palsy, Muscular Dystrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Andrew Bush, Professor principal investigator
Imperial College London
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing saline in people with Neuromuscular Diseases and Neurodevelopmental Disorders, run by Imperial College London at 2 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 15 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Natalia G Galaz Souza (0787131892, natalia.galaz-souza16@imperial.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Course of antibiotics for respiratory infections (time frame: from baseline to week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2024.