Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
5372
Study information
Scientific title
Evaluation of the impact of a CoughAssist® mechanical in-exsufflator (MI-E) device on morbidity, quality of life and survival in patients with motor neurone disease (MND) using non-invasive ventilation (NIV)
Acronym
Evaluating CoughAssist
Study hypothesis
Without active management of respiratory symptoms, the majority of patients with motor neurone disease (MND) die within approximately 3 - 5 years, from neuromuscular respiratory failure. The applicants have previously shown that non-invasive ventilation (NIV) has a significant positive effect on the quality of life as well as prolonging the survival of MND patients, particularly in patients without severe bulbar dysfunction. However, increasing life expectancy by starting patients on NIV can uncover other downstream problems and a major problem for the patient is the inability to cough effectively. Poor cough can result in chest infections and airway blockage and pulmonary collapse. These problems lead to increasing respiratory failure and often require repeated hospital admissions.
The aim of the present study is to determine whether the use of a mechanical CoughAssist® device can reduce the symptoms of chest infections and therefore hospital admissions whilst improving the quality of life and survival of patients with MND who need to be started on using non-invasive ventilation (NIV). 40 MND patients who require NIV will be assigned to one of two groups via a process of minimisation. One cohort will be assigned to receive NIV and be taught how to use breath-stacking. The other cohort will be assigned to receive NIV and be given the use of a mechanical in-exsufflator (MI-E) (CoughAssist®) machine. Patients will be reviewed every 3 months for 1 year.
Outcome measures include the number of days with symptoms of chest infection, number of days in hospital due to chest infection, quality of life measures, measures of respiratory and neuromuscular function, survival of the patient and the impact of the intervention on the carer. Power calculations are based on the best information available in the literature regarding the number of days of hospitalisation expected for MND patients started on NIV. All outcome measures will be analysed by intention to treat; tests of significance will be two-sided.
Ethics approval
MREC approved, ref: 08/H1313/83
Study design
Single-centre randomised interventional prevention, process of care and treatment trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Other
Trial type
Treatment
Patient information sheet
Condition
Topic: Dementias and Neurodegenerative Diseases Research Network; Subtopic: Motor neurone disease; Disease: Motor neurone disease
Intervention
CoughAssist® mechanical in-exsufflator versus breath stacking technique.
Study entry: single randomisation only
Intervention type
Device
Phase
Not Applicable
Drug names
Primary outcome measure
Number of days with symptoms of chest infections requiring antibiotic therapy
Secondary outcome measures
1. Number of days in as a hospital inpatient due to chest infection
2. Quality of life (36-item short form health survey [SF-36])
Overall trial start date
26/05/2009
Overall trial end date
01/11/2011
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. The patient should not have any serious medical problems, apart from MND, which may reduce life expectancy
2. The patient should be capable of giving informed consent and should not have evidence of any significant impairment of cognitive function
3. Patients with MND in respiratory failure meeting two of the following criteria indicating the need for NIV:
3.1. Nocturnal or daytime hypercapnia (arterial blood gas [ABG] partial pressure of carbon dioxide in the blood [PaCO2] greater than 6.0 kPa)
3.2. Nocturnal hypoxaemia (oxygen saturation [SaO2] less than 88% for 5 consecutive minutes of the time asleep)
3.3. Lung function tests - forced vital capacity (FVC) less than 60% predicted
3.4. Maximal expiratory pressure less than 60 cm H2O
3.5. Orthopnoea
3.6. Symptoms of hypersomnolence or non-refreshing sleep
4. The patient should have a main carer who is willing to assist the patient in following the treatment regimen
5. The patient must fulfill the El Escorial clinically or laboratory supported probable or definite criteria for MND
Participant type
Patient
Age group
Adult
Gender
Not Specified
Target number of participants
Planned sample size: 40; UK sample size: 40
Participant exclusion criteria
1. Inability to tolerate NIV
2. Infringement of the contraindications of CoughAssist® (a history of bullous emphysema, susceptibility to pneumothorax or pneumo-mediastinum, or recent baratrauma)
3. Presence of a significant medical condition, other than MND, which may reduce life expectancy
4. Presence of significant impairment of cognitive function (for example, clinically overt fronto-temporal dysfunction which is clinically evident and noticeable to the family)
5. Participation in any other interventional trial
Recruitment start date
26/05/2009
Recruitment end date
01/11/2011
Locations
Countries of recruitment
United Kingdom
Trial participating centre
Royal Hallamshire Hospital
Sheffield
S10 2JF
United Kingdom
Sponsor information
Organisation
Sheffield Teaching Hospitals NHS Trust (UK)
Sponsor details
Royal Hallamshire Hospital
Glossop Road
Sheffield
S10 2JF
United Kingdom
Sponsor type
Hospital/treatment centre
Website
Funders
Funder type
Research organisation
Funder name
Motor Neurone Disease Association (UK) (ref: shaw/Apr08/6335)
Alternative name(s)
MNDA
Funding Body Type
private sector organisation
Funding Body Subtype
professional associations and societies
Location
United Kingdom
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Basic results (scientific)
Publication list
2015 results in https://www.ncbi.nlm.nih.gov/pubmed/26140500