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Nebulised Hypertonic Saline in Children and Young People With Neuromuscular Disease and Cerebral Palsy

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ClinicalTrials.gov Identifier: NCT03623698
Recruitment Status : Recruiting
First Posted : August 9, 2018
Last Update Posted : August 9, 2018
Sponsor:
Collaborator:
Royal Brompton & Harefield NHS Foundation Trust
Information provided by (Responsible Party):
Imperial College London

Brief 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. 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.

Condition or disease Intervention/treatment
Neuromuscular Diseases Cerebral Palsy Drug: Nebulised hypertonic saline

  Show Detailed Description

Study Type : Observational
Estimated Enrollment : 40 participants
Observational Model: Case-Only
Time Perspective: Retrospective
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
Actual Study Start Date : June 15, 2018
Estimated Primary Completion Date : September 2018
Estimated Study Completion Date : December 2018

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
Neuromuscular disease

Children and young people with neuromuscular disease that have been on treatment with nebulised hypertonic saline for at least 12 months.

2 questionnaires will be applied to their parent or legal guardian and one questionnaire will be applied to children aged 10 years or older.

Drug: Nebulised hypertonic saline
Nebulised hypertonic saline used for a period of at least 12 months
Other Names:
  • 3% Sodium Chloride
  • 5% Sodium Chloride
  • 7% Sodium Chloride

Cerebral Palsy

Children and young people with cerebral palsy that have been on treatment with nebulised hypertonic saline for at least 12 months.

2 questionnaires will be applied to their parent or legal guardian.

Drug: Nebulised hypertonic saline
Nebulised hypertonic saline used for a period of at least 12 months
Other Names:
  • 3% Sodium Chloride
  • 5% Sodium Chloride
  • 7% Sodium Chloride




Primary Outcome Measures :
  1. Number of respiratory exacerbations [ Time Frame: Change from baseline number of annual exacerbations requiring antibiotic treatment at 12 months after starting hypertonic saline treatment ]
    Number of respiratory exacerbations requiring antibiotic treatment


Secondary Outcome Measures :
  1. Number of hospitalsations due to respiratory exacerbations [ Time Frame: Change from baseline number of annual hospitalisations at 12 months after starting hypertonic saline ]
    Questionnaire

  2. Participant's perception of treatment [ Time Frame: At 12 months after starting treatment with hypertonic saline ]
    Questionnaire: "Hypertonic saline treatment questionnaire". Applied to all participants aged 10-15 years. Perception of overall usefulness of nebulised hypertonic saline: "Useful", "Not useful", "I don't know".

  3. Parent's or legal guardian's perception of treatment [ Time Frame: At 12 months after starting treatment with hypertonic saline ]
    "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.

  4. Score on the ease of airway clearance pictorial analogue scale from children and young adults as participants [ Time Frame: Change from baseline score on ease of airway clearance at 12 months after starting treatment with hypertonic saline ]
    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.

  5. Score on the ease of airway clearance from legal guardian [ Time Frame: Change from baseline score on ease of airway clearance at 12 months after starting treatment with hypertonic saline ]
    Measures ease of airway clearance perceived by legal guardian through a likert scale on easy of airway clearance: 1) Very easy, 2) Easy, 3) Neither easy nor difficult, 4) Not easy, 5) Not at all easy.

  6. Nocturnal apnea index [ Time Frame: Change from baseline nocturnal apnea index at 12 months after starting treatment with hypertonic saline ]
    Apnea index

  7. Nocturnal hipopnea index [ Time Frame: Change from baseline nocturnal hipopnea index at 12 months after starting treatment with hypertonic saline ]
    Hipopnea index

  8. Nocturnal oxygen saturation [ Time Frame: Change from baseline nocturnal oxygen saturation at 12 months after starting treatment with hypertonic saline ]
    %SpO2

  9. Nocturnal oxygen desaturation index (ODI) [ Time Frame: Change from baseline nocturnal oxygen desaturation index at 12 months after starting treatment with hypertonic saline ]
    Number of desaturations per hour of sleep

  10. Nocturnal Transcutaneous Carbon Dioxide (TcPCO2) [ Time Frame: Change from baseline nocturnal transcutaneous carbon dioxide at 12 months after starting treatment with hypertonic saline ]
    TcPCO2 (mmHg)

  11. Rate of decline of Tiffenau index [ Time Frame: Change from baseline Tiffenau index at 12 months after starting treatment with hypertonic saline ]
    FEV1/FVC

  12. Rate of decline of Forced Expiratory Volume in first second (FEV1) percentage of predicted [ Time Frame: Change from baseline forced expiratory volume at first second at 12 months after starting treatment with hypertonic saline ]
    FEV1% predicted

  13. Rate of decline of Forced Vital Capacity (FVC) percentage of predicted [ Time Frame: Change from baseline forced vital capacity at 12 months after starting treatment with hypertonic saline ]
    FVC% predicted

  14. Rate of decline of Peak Expiratory Flow (PEF) [ Time Frame: Change from baseline peak expiratory flow at 12 months after starting treatment with hypertonic saline ]
    litres/minute



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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Sampling Method:   Non-Probability Sample
Study Population
Children with Neuromuscular disease or Cerebral Palsy who have their care at the Royal Brompton Hospital.
Criteria

Inclusion Criteria:

  • Children with Neuromuscular disease or Cerebral Palsy who have been on treatment with nebulised Hypertonic Saline for at least 12 months.

Exclusion Criteria:

  • Children also diagnosed with cystic fibrosis.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03623698


Contacts
Contact: Hui Leng Tan, Doctor +442073528121 h.tan@rbht.nhs.uk
Contact: Natalia Galaz Souza, Professional +44744050834 natalia.galaz-souza16@imperial.ac.uk

Locations
United Kingdom
Royal Brompton Hospital Recruiting
London, United Kingdom, SW36NP
Contact: Hui Leng Tan, Doctor    +442073528121    h.tan@rhbt.nhs.uk   
Contact: Natalia Galaz Souza    +447440508344    natalia.galaz-souza16@imperial.ac.uk   
Sub-Investigator: Andrew Bush, Professor         
Principal Investigator: Natalia Galaz Souza, Professional         
Sponsors and Collaborators
Imperial College London
Royal Brompton & Harefield NHS Foundation Trust
Investigators
Principal Investigator: Natalia Galaz Souza, Professional Imperial College London

Publications:

Responsible Party: Imperial College London
ClinicalTrials.gov Identifier: NCT03623698     History of Changes
Other Study ID Numbers: 18IC4403
First Posted: August 9, 2018    Key Record Dates
Last Update Posted: August 9, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: Data ownership rights will lie with the institution.

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Imperial College London:
Hypertonic saline
nebulised
children
young people
adolescents
respiratory

Additional relevant MeSH terms:
Cerebral Palsy
Neuromuscular Diseases
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases