Neuromuscular DiseasesNCT03623698
Nebulised Hypertonic Saline in Children and Young People With Neuromuscular Disease and Cerebral Palsy
An observational study of Neuromuscular Diseases, run by Imperial College London at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Neuromuscular Diseases
- What is being tested?
- Nebulised hypertonic saline
- How many people
- 24 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 15 Jun 2018to 26 Sep 2019
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 1 year to 18 years
- All sexes
- Child, Adult
Study population: Children with Neuromuscular disease or Cerebral Palsy who have their care at the Royal Brompton Hospital.
Who may be able to join
- Children with Neuromuscular disease or Cerebral Palsy who have been on treatment with nebulised Hypertonic Saline for at least 12 months.
Who cannot join
- Children also diagnosed with cystic fibrosis.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Chart Review Assessing the Effects of Nebulised Hypertonic Saline on Respiratory-related Complications in Children and Young People With Neuromuscular Disease and Cerebral Palsy
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.
Read the detailed description
Chart review of children and young people with Neuromuscular disease or Cerebral Palsy who are cared for in the Royal Brompton Hospital and that have been treated with nebulised hypertonic saline for at least 12 months.
To further complement data from hospital records, two questionnaires will be applied. Parents of children who meet criteria will be asked to complete the following questionnaires:
- The National Health and Nutrition Examination Survey (NHANES) for Hospitalisation and access to are - HUQ.010; and
- Questionnaire on Hypertonic Saline treatment.
Children from 10 - 18 years will be asked to complete the Questionnaire on Hypertonic Saline treatment.
AIMS
- Explore whether treatment with nebulised Hypertonic Saline in children with Neuromuscular disease or Cerebral Palsy decreases respiratory-related complications.
- Evaluate whether the treatment with nebulised hypertonic saline in children with neuromuscular disease or cerebral palsy improves the ease of airway clearance.
- Explore how parents of children with Neuromuscular disease and children with Cerebral Palsy perceive the treatment with nebulised hypertonic saline compared with previous management.
Sample Size:
The investigators aim to recruit 40 participants for each group, including children and young people and their parents or legal guardians, as this is a pilot study.
STATISTICAL ANALYSIS PLAN
- Univariate X2 analysis for categorical variables to investigate Courses of antibiotic treatment.
- Univariate X2 analysis for categorical variables to investigate Number of hospitalisations.
- Student t testing will be used to analyse nocturnal oxygenation and ventilation outcomes comparing one year before and after starting treatment with Hypertonic Saline.
- Independent t testing and Mann-Whitney U test to analyse Rate of decline in pulmonary function.
- Cox proportional hazard model to test differences in primary endpoints for different baseline FVC.
- Univarate analysis to analyse Ease of airway clearance.
- Univariate analysis on perception of treatment.
Data and all appropriate documentation will be stored for a minimum of 10 years after the completion of the study, including the follow-up period.
ETHICS APPROVAL The Study Coordination Centre has obtained approval from the Yorkshire \& The Humber - Leeds West Research Ethics Committee (REC) and Health Regulatory Authority (HRA). The study also received confirmation of capacity and capability from each participating NHS Trust before accepting participants into the study or any research activity was carried out. The study will be conducted in accordance with the recommendations for physicians involved in research on human subjects adopted by the 18th World Medical Assembly, Helsinki 1964 and later revisions.
CONSENT Consent to enter the study must be sought from each participant only after a full explanation has been given, an information leaflet offered and time allowed for consideration. Signed participant consent should be obtained. The right of the participant to refuse to participate without giving reasons must be respected. After the participant has entered the study the clinician remains free to give alternative treatment to that specified in the protocol at any stage if he/she feels it is in the participant's best interest, but the reasons for doing so should be recorded. In these cases the participants remain within the study for the purposes of follow-up and data analysis. All participants are free to withdraw at any time from the protocol treatment without giving reasons and without prejudicing further treatment.
CONFIDENTIALITY The Chief Investigator will preserve the confidentiality of participants taking part in the study and is registered under the Data Protection Act.
PUBLICATION POLICY Data ownership rights will lie with the institution. Findings of this study will be presented as a Dissertation and will be available through Open Access. The investigators aim to publish findings in peer-review journals.
- Conditions
- Neuromuscular Diseases
- Being tested
- Nebulised hypertonic saline
- Keywords
- Hypertonic saline, nebulised, children, young people, adolescents, respiratory
- Medical subject headings
- Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nebulised hypertonic salineDrug
Also called 3% Sodium Chloride, 5% Sodium Chloride, 7% Sodium Chloride
Nebulised hypertonic saline used for a period of at least 12 months
| Study group | Role | What they receive |
|---|---|---|
| Before treatment | Children and young people with neuromuscular disease during the 12 months before being prescribed treatment with nebulised saline (0.9% - 7%) | |
| After treatment | Children and young people with neuromuscular disease during the 12 months after being prescribed treatment with nebulised saline (0.9% - 7%) Receives: Drug: Nebulised hypertonic saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Antibiotic Courses
Treatments due to respiratory exacerbations
Measured: Change from baseline (before treatment) and 12 months after treatment
Number of Hospitalsations Due to Respiratory Exacerbations
Number of respiratory exacerbations that required not planned hospitalisation
Measured: Change from baseline (before treatment) and 12 months after treatment
Secondary outcomes (13)
Participant's Perception of Treatment
Questionnaire: "Hypertonic saline treatment questionnaire". Perception of overall usefulness of nebulised hypertonic saline: "Useful", "Not useful", "I don't know".
Measured: At 12 months after starting treatment with hypertonic saline
Parent's or Legal Guardian's Perception of Treatment
"Hypertonic saline treatment questionnaire for legal guardian". Measures the perception of overall usefulness of nebulised hypertonic saline through a likert scale: Very useful, useful, neither useful or not useful, not useful, not at all useful.
Measured: At 12 months after starting treatment with hypertonic saline
Score on the Ease of Airway Clearance Pictorial Analogue Scale From Children and Young Adults as Participants
Pictorial visual scale "Facial Rating of perceived exertion Scale". Measures ease of airway clearance. Values range starting in 0 (Extremely easy) to 10 (Extremely hard), including 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10. Numbers are also associated with facial expressions.
Measured: Change from baseline (before treatment) and 12 months after treatment
Score on the Ease of Airway Clearance From Parents or Legal Guardians
Measures ease of airway clearance through a 1-5 likert scale: 1) Very easy, 2) Easy, 3) Neither easy nor difficult, 4) Not easy, 5) Not at all easy.
Measured: Change from baseline (before treatment) and 12 months after treatment
Apnea Index (AI)
The number of apneas recorded during the study per hour of sleep
Measured: Change from baseline (before treatment) and 12 months after treatment
AHI
Nocturnal Apnoea Hipopnea index: total number of apnea events plus hypopnea events divided by the total number of minutes of actual sleep time and then multiplied by 60.
Measured: Change from baseline (before treatment) and 12 months after treatment
%SpO2
Nocturnal oxygen saturation
Measured: Change from baseline (before treatment) and 12 months after treatment
Nocturnal ODI
Oxygen desaturation index: Number of desaturations per hour of sleep
Measured: Change from baseline (before treatment) and 12 months after treatment
TcPCO2
Nocturnal Transcutaneous Carbon Dioxide in kPa
Measured: Change from baseline (before treatment) and 12 months after treatment
FEV1/FVC %Predicted Rate of Decline
Rate of decline per year of Tiffenau index
Measured: Change from baseline (before treatment) and 12 months after treatment
FEV1% Predicted Rate of Decline
Rate of decline of Forced Expiratory Volume in first second (FEV1) percentage of predicted.
Rate of decline is a measure of slope of FEV1 percentage predicted.
Baseline slope: [(FEV1% at baseline / FEV1% 12 months before treatment) - 1] * 100 After treatment slope: [(FEV1% 12 months after treatment / FEV1% at baseline) - 1] * 100
Measured: Change from the baseline (before treatment) and 12 months after treatment
FVC% Predicted Rate of Decline
Rate of decline of Forced Vital Capacity (FVC) percentage of predicted. Rate of decline is a measure of slope of FVC%.
Baseline slope: [(FVC% at baseline / FVC% 12 months before treatment) - 1] * 100 After treatment slope: [(FVC% 12 months after treatment / FVC% at baseline) - 1] * 100
Measured: Change from baseline (before treatment) and 12 months after treatment
Peak Expiratory Flow (PEF)
Peak expiratory flow percentage of predicted
Measured: Change from baseline peak expiratory flow at 12 months after starting treatment with hypertonic saline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2018
- Study start15 Jun 2018
- Primary completion14 Aug 2019
- Study completion26 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (11)Papers the sponsor has linked to this study.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Imperial College London
- Sponsor type
- University, hospital or other
- Collaborators
- Royal Brompton & Harefield NHS Foundation Trust
- Responsible party
- Sponsor
- Investigators
- Natalia Galaz Souza, Professional, Principal investigator, Imperial College London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03623698
- Sponsor's protocol ID
- 18IC4403
- Status last verified
- Aug 2020
- First submitted
- 16 Jul 2018
- Last updated
- 16 Sep 2020
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Data ownership rights will lie with the institution.
Publications
Allen J. Pulmonary complications of neuromuscular disease: a respiratory mechanics perspective. Paediatr Respir Rev. 2010 Mar;11(1):18-23. doi: 10.1016/j.prrv.2009.10.002. Epub 2009 Dec 2.
PubMed 20113987Lo Mauro A, Aliverti A. Physiology of respiratory disturbances in muscular dystrophies. Breathe (Sheff). 2016 Dec;12(4):318-327. doi: 10.1183/20734735.012716.
PubMed 28210319Boitano LJ. Management of airway clearance in neuromuscular disease. Respir Care. 2006 Aug;51(8):913-22; discussion 922-4.
PubMed 16867201Gerdung CA, Tsang A, Yasseen AS 3rd, Armstrong K, McMillan HJ, Kovesi T. Association Between Chronic Aspiration and Chronic Airway Infection with Pseudomonas aeruginosa and Other Gram-Negative Bacteria in Children with Cerebral Palsy. Lung. 2016 Apr;194(2):307-14. doi: 10.1007/s00408-016-9856-5. Epub 2016 Feb 16.
PubMed 26883134Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2001 Dec 15;164(12):2191-4. doi: 10.1164/ajrccm.164.12.2103052.
PubMed 11751186Bell CF, Kurosky SK, Candrilli SD. Muscular dystrophy-related hospitalizations among male pediatric patients in the United States. Hosp Pract (1995). 2015;43(3):180-5. doi: 10.1080/21548331.2015.1033375. Epub 2015 Apr 1.
PubMed 25833749Yuan JX, McGowan M, Hadjikoumi I, Pant B. Do children with neurological disabilities use more inpatient resources: an observational study. Emerg Themes Epidemiol. 2017 Apr 27;14:5. doi: 10.1186/s12982-017-0059-1. eCollection 2017.
PubMed 28465710Elkins MR, Bye PT. Mechanisms and applications of hypertonic saline. J R Soc Med. 2011 Jul;104 Suppl 1(Suppl 1):S2-5. doi: 10.1258/jrsm.2011.s11101. No abstract available.
PubMed 21719889Elkins MR, Robinson M, Rose BR, Harbour C, Moriarty CP, Marks GB, Belousova EG, Xuan W, Bye PT; National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med. 2006 Jan 19;354(3):229-40. doi: 10.1056/NEJMoa043900.
PubMed 16421364Amin R, Subbarao P, Jabar A, Balkovec S, Jensen R, Kerrigan S, Gustafsson P, Ratjen F. Hypertonic saline improves the LCI in paediatric patients with CF with normal lung function. Thorax. 2010 May;65(5):379-83. doi: 10.1136/thx.2009.125831.
PubMed 20435858Galaz Souza N, Bush A, Tan HL. Exploratory study of the effectiveness of nebulised saline in children with neurodisability. Eur Respir J. 2021 Mar 18;57(3):2001407. doi: 10.1183/13993003.01407-2020. Print 2021 Mar.
PubMed 33184120
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Chart Review Assessing the Effects of Nebulised Hypertonic Saline on Respiratory-related Complications in Children and Young People With Neuromuscular Disease and Cerebral Palsy
- ClinicalTrials.gov ID
- NCT03623698
- Acronym / sponsor ID
- 18IC4403
- Conditions
- Neuromuscular Diseases
- Keywords
- Hypertonic saline, nebulised, children, young people, adolescents, respiratory
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Imperial College LondonUniversity, hospital or other
- Collaborators
- Royal Brompton & Harefield NHS Foundation Trust
- Study start
- 15 Jun 2018 (actual)
- Primary completion
- 14 Aug 2019 (actual)
- Study completion
- 26 Sep 2019 (actual)
- First posted
- 9 Aug 2018 (actual)
- Results
- Posted 16 Sep 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Chart review of children and young people with Neuromuscular disease or Cerebral Palsy who are cared for in the Royal Brompton Hospital and that have been treated with nebulised hypertonic saline for at least 12 months.
To further complement data from hospital records, two questionnaires will be applied. Parents of children who meet criteria will be asked to complete the following questionnaires:
- The National Health and Nutrition Examination Survey (NHANES) for Hospitalisation and access to are - HUQ.010; and
- Questionnaire on Hypertonic Saline treatment.
Children from 10 - 18 years will be asked to complete the Questionnaire on Hypertonic Saline treatment.
AIMS
- Explore whether treatment with nebulised Hypertonic Saline in children with Neuromuscular disease or Cerebral Palsy decreases respiratory-related complications.
- Evaluate whether the treatment with nebulised hypertonic saline in children with neuromuscular disease or cerebral palsy improves the ease of airway clearance.
- Explore how parents of children with Neuromuscular disease and children with Cerebral Palsy perceive the treatment with nebulised hypertonic saline compared with previous management.
Sample Size:
The investigators aim to recruit 40 participants for each group, including children and young people and their parents or legal guardians, as this is a pilot study.
STATISTICAL ANALYSIS PLAN
- Univariate X2 analysis for categorical variables to investigate Courses of antibiotic treatment.
- Univariate X2 analysis for categorical variables to investigate Number of hospitalisations.
- Student t testing will be used to analyse nocturnal oxygenation and ventilation outcomes comparing one year before and after starting treatment with Hypertonic Saline.
- Independent t testing and Mann-Whitney U test to analyse Rate of decline in pulmonary function.
- Cox proportional hazard model to test differences in primary endpoints for different baseline FVC.
- Univarate analysis to analyse Ease of airway clearance.
- Univariate analysis on perception of treatment.
Data and all appropriate documentation will be stored for a minimum of 10 years after the completion of the study, including the follow-up period.
ETHICS APPROVAL The Study Coordination Centre has obtained approval from the Yorkshire \& The Humber - Leeds West Research Ethics Committee (REC) and Health Regulatory Authority (HRA). The study also received confirmation of capacity and capability from each participating NHS Trust before accepting participants into the study or any research activity was carried out. The study will be conducted in accordance with the recommendations for physicians involved in research on human subjects adopted by the 18th World Medical Assembly, Helsinki 1964 and later revisions.
CONSENT Consent to enter the study must be sought from each participant only after a full explanation has been given, an information leaflet offered and time allowed for consideration. Signed participant consent should be obtained. The right of the participant to refuse to participate without giving reasons must be respected. After the participant has entered the study the clinician remains free to give alternative treatment to that specified in the protocol at any stage if he/she feels it is in the participant's best interest, but the reasons for doing so should be recorded. In these cases the participants remain within the study for the purposes of follow-up and data analysis. All participants are free to withdraw at any time from the protocol treatment without giving reasons and without prejudicing further treatment.
CONFIDENTIALITY The Chief Investigator will preserve the confidentiality of participants taking part in the study and is registered under the Data Protection Act.
PUBLICATION POLICY Data ownership rights will lie with the institution. Findings of this study will be presented as a Dissertation and will be available through Open Access. The investigators aim to publish findings in peer-review journals.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Before treatment | – | –Children and young people with neuromuscular disease during the 12 months before being prescribed treatment with nebulised saline (0.9% - 7%) |
| After treatment | – | Drug: Nebulised hypertonic salineChildren and young people with neuromuscular disease during the 12 months after being prescribed treatment with nebulised saline (0.9% - 7%) |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- Children with Neuromuscular disease or Cerebral Palsy who have their care at the Royal Brompton Hospital.
Key inclusion criteria (1 listed)
- Children with Neuromuscular disease or Cerebral Palsy who have been on treatment with nebulised Hypertonic Saline for at least 12 months.
Key exclusion criteria (1 listed)
- Children also diagnosed with cystic fibrosis.
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 |
|---|---|---|
| Nebulised hypertonic salineAlso: 3% Sodium Chloride, 5% Sodium Chloride, 7% Sodium ChlorideNebulised hypertonic saline used for a period of at least 12 months | Drug | After treatment |
Primary outcome
Change in Antibiotic Courses
Time frame: Change from baseline (before treatment) and 12 months after treatment
Treatments due to respiratory exacerbations
Number of Hospitalsations Due to Respiratory Exacerbations
Time frame: Change from baseline (before treatment) and 12 months after treatment
Number of respiratory exacerbations that required not planned hospitalisation
13 secondary outcomes
Participant's Perception of Treatment
Time frame: At 12 months after starting treatment with hypertonic saline
Parent's or Legal Guardian's Perception of Treatment
Time frame: At 12 months after starting treatment with hypertonic saline
Score on the Ease of Airway Clearance Pictorial Analogue Scale From Children and Young Adults as Participants
Time frame: Change from baseline (before treatment) and 12 months after treatment
Score on the Ease of Airway Clearance From Parents or Legal Guardians
Time frame: Change from baseline (before treatment) and 12 months after treatment
Apnea Index (AI)
Time frame: Change from baseline (before treatment) and 12 months after treatment
AHI
Time frame: Change from baseline (before treatment) and 12 months after treatment
%SpO2
Time frame: Change from baseline (before treatment) and 12 months after treatment
Nocturnal ODI
Time frame: Change from baseline (before treatment) and 12 months after treatment
TcPCO2
Time frame: Change from baseline (before treatment) and 12 months after treatment
FEV1/FVC %Predicted Rate of Decline
Time frame: Change from baseline (before treatment) and 12 months after treatment
FEV1% Predicted Rate of Decline
Time frame: Change from the baseline (before treatment) and 12 months after treatment
FVC% Predicted Rate of Decline
Time frame: Change from baseline (before treatment) and 12 months after treatment
Peak Expiratory Flow (PEF)
Time frame: Change from baseline peak expiratory flow at 12 months after starting treatment with hypertonic saline
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 20113987Allen J. Pulmonary complications of neuromuscular disease: a respiratory mechanics perspective. Paediatr Respir Rev. 2010 Mar;11(1):18-23. doi: 10.1016/j.prrv.2009.10.002. Epub 2009 Dec 2.
- Background: PubMed 28210319Lo Mauro A, Aliverti A. Physiology of respiratory disturbances in muscular dystrophies. Breathe (Sheff). 2016 Dec;12(4):318-327. doi: 10.1183/20734735.012716.
- Background: PubMed 16867201Boitano LJ. Management of airway clearance in neuromuscular disease. Respir Care. 2006 Aug;51(8):913-22; discussion 922-4.
- Background: PubMed 26883134Gerdung CA, Tsang A, Yasseen AS 3rd, Armstrong K, McMillan HJ, Kovesi T. Association Between Chronic Aspiration and Chronic Airway Infection with Pseudomonas aeruginosa and Other Gram-Negative Bacteria in Children with Cerebral Palsy. Lung. 2016 Apr;194(2):307-14. doi: 10.1007/s00408-016-9856-5. Epub 2016 Feb 16.
- Background: PubMed 11751186Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2001 Dec 15;164(12):2191-4. doi: 10.1164/ajrccm.164.12.2103052.
- Background: PubMed 25833749Bell CF, Kurosky SK, Candrilli SD. Muscular dystrophy-related hospitalizations among male pediatric patients in the United States. Hosp Pract (1995). 2015;43(3):180-5. doi: 10.1080/21548331.2015.1033375. Epub 2015 Apr 1.
- Background: PubMed 28465710Yuan JX, McGowan M, Hadjikoumi I, Pant B. Do children with neurological disabilities use more inpatient resources: an observational study. Emerg Themes Epidemiol. 2017 Apr 27;14:5. doi: 10.1186/s12982-017-0059-1. eCollection 2017.
- Background: PubMed 21719889Elkins MR, Bye PT. Mechanisms and applications of hypertonic saline. J R Soc Med. 2011 Jul;104 Suppl 1(Suppl 1):S2-5. doi: 10.1258/jrsm.2011.s11101. No abstract available.
- Background: PubMed 16421364Elkins MR, Robinson M, Rose BR, Harbour C, Moriarty CP, Marks GB, Belousova EG, Xuan W, Bye PT; National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med. 2006 Jan 19;354(3):229-40. doi: 10.1056/NEJMoa043900.
- Background: PubMed 20435858Amin R, Subbarao P, Jabar A, Balkovec S, Jensen R, Kerrigan S, Gustafsson P, Ratjen F. Hypertonic saline improves the LCI in paediatric patients with CF with normal lung function. Thorax. 2010 May;65(5):379-83. doi: 10.1136/thx.2009.125831.
- Derived from this study: PubMed 33184120Galaz Souza N, Bush A, Tan HL. Exploratory study of the effectiveness of nebulised saline in children with neurodisability. Eur Respir J. 2021 Mar 18;57(3):2001407. doi: 10.1183/13993003.01407-2020. Print 2021 Mar.
- Individual participant dataSharing planned: Undecided. Data ownership rights will lie with the institution.
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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. This study is not recruiting at the moment.
Natalia Galaz Souza, Professional principal investigator
Imperial College London
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neuromuscular Diseases, run by Imperial College London at 1 site 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 1 inclusion and 1 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: finished, as of the sponsor's last update on 16 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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?
Yes. Results are posted in the registry (first posted 16 Sep 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Antibiotic Courses (time frame: Change from baseline (before treatment) and 12 months after treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03623698, which also lists 11 linked publications.
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 16 Sep 2020.