Impact of In-exsufflator Treatment on hospitalisation for Respiratory Exacerbation in Neuromuscular Disease

ISRCTN ISRCTN21996121
DOI https://doi.org/10.1186/ISRCTN21996121
Secondary identifying numbers N/A
Submission date
26/06/2007
Registration date
13/09/2007
Last edited
07/11/2007
Recruitment status
No longer recruiting
Overall study status
Completed
Condition category
Nervous System Diseases
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr Sherri Katz
Scientific

Children's Hospital of Eastern Ontario
401 Smyth Road
w1406
Ottawa
K1H 8L1
Canada

+1 613 737 7600 ext. 2868
skatz@cheo.on.ca

Study information

Study designMulti-centre randomised unblinded controlled trial of the mechanical inexsufflator. Randomisation sequence will be stratified by centre with a block-size randomisation protocol.
Primary study designInterventional
Secondary study designRandomised controlled trial
Study setting(s)Hospital
Study typeTreatment
Scientific title
Study acronymIn-ex TREND study
Study objectivesWe will be looking specifically at a heterogeneous group of neuromuscular disorders which, either as a secondary consequence of degeneration of the spinal nerves (spinal muscular atrophy, amyotrophic lateral sclerosis) or as a primary muscle defect (muscular dystrophies, myopathies) result in progressive loss of muscle strength. Respiratory complications are the primary cause of morbidity and mortality associated with these diseases, as involvement of the respiratory muscles leads to either progressive hypoventilation or recurrent atelectasis and pneumonia secondary to decreased cough efficacy.

For this study we will look at those children with neuromuscular disorders who are admitted to hospital with a respiratory deterioration (hypoxemia and/or the presence of new onset radiologically proven atelectasis or consolidation).

Hypotheses:
We expect that the addition of the Emerson in-exsufflator to a standard treatment regimen for acute respiratory deterioration:
1. Will result in a decreased duration of hospitalisation in a population of children with neuromuscular disease
2. Resulting in hospitalisation will decrease the time requiring supplemental oxygen in a population of children with neuromuscular disease
3. Resulting in hospitalisation will result in a more rapid improvement in chest X-ray changes in a population of children with neuromuscular disease
4. Resulting in hospitalisation will decrease the length of stay in intensive care unit or days invasively ventilated in a population of children with neuromuscular disease
Ethics approval(s)Each site will submit to their local hospital ethics boards. Ethics approval received from the Children's Hospital of Eastern Ontario Research Ethics Board (REB) on the 2nd October 2007 (ref: 07/24E).
Health condition(s) or problem(s) studiedChildren with neuromuscular disorders who are admitted to hospital with a respiratory deterioration (hypoxemia and/or the presence of new onset radiologically proven atelectasis or consolidation).
Intervention1. Conventional treatments, as deemed appropriate by the treating physician:
1.1. Physiotherapy
1.2. Nutritional support
1.3. Antibiotics (fever, elevated White Blood Cells [WBC])
1.4. Non-invasive positive pressure ventilation
2. Conventional treatments and Emerson in-exsufflator

Using Friedman's formula for survival analysis study design, 62 patients per arm would achieve 80% power to detect a hazard ratio of 1.4. To account for potential withdrawals and withdrawal of consent, estimated at about 2.5%, four additional participants will be recruited for a grand total of 128 participants.
Intervention typeOther
Primary outcome measureTime to discharge: an estimate of the primary end-point, time to discharge with standard care, was based on the clinical experience of the principal investigators and is currently being verified with a three-year retrospective chart review at Childrens Hospital of Eastern Ontario (CHEO). The mean length of stay in these patients is estimated to be 10 days. Discussions with several paediatric respirologists have taken place, focusing on what magnitude of difference in time to discharge would be clinically important between treatment and control groups. The consensus was a Minimally Clinically Important Difference (MCID) of three days' reduction from the average current length of stay in the study population. These numbers translate to a hazard ratio of 1.4. A two-sided time-to-event test at a = 0.05 will be used to detect a significant difference in time to discharge between the two arms.
Secondary outcome measures1. Time (in days) to improvement in oxygenation (no longer requiring supplemental oxygen for 24 hours)
2. X-ray changes: improvement or progression (increasing atelectasis, consolidation)
3. Development of acute hypercapnic respiratory failure requiring intubation and mechanical ventilation
4. Days in intensive care unit
Overall study start date01/10/2007
Completion date01/04/2009

Eligibility

Participant type(s)Patient
Age groupChild
Lower age limit3 Years
Upper age limit17 Years
SexAll
Target number of participants128
Key inclusion criteria1. Patients aged 3 to 17 years
2. Patients have a known neuromotor disorder affecting respiratory muscles
3. Admitted to hospital with a respiratory deterioration (hypoxemia in the presence of new onset radiologically proven atelectasis or consolidation)
Key exclusion criteria1. Refusal to participate
2. Already using the Emerson in-exsufflator at home on a regular basis
3. Development of new uncompensated hypercapnic respiratory failure requiring intubation and mechanical ventilation
4. History of bullous emphysema, known susceptibility to pneumothorax or pneumomediastinum, or known to have had any recent barotraumas
Date of first enrolment01/10/2007
Date of final enrolment01/04/2009

Locations

Countries of recruitment

  • Canada

Study participating centre

Children's Hospital of Eastern Ontario
Ottawa
K1H 8L1
Canada

Sponsor information

Funders

Funder type

Charity

Fight Spinal Muscular Atrophy (FightSMA) (USA)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to shareNo
IPD sharing plan summaryNot provided at time of registration
Publication and dissemination planNot provided at time of registration
IPD sharing plan
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