Condition category
Nervous System Diseases
Date applied
26/06/2007
Date assigned
13/09/2007
Last edited
07/11/2007
Prospective/Retrospective
Prospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr Sherri Katz

ORCID ID

Contact details

Children's Hospital of Eastern Ontario
401 Smyth Road
w1406
Ottawa
K1H 8L1
Canada
+1 613 737 7600 ext. 2868
skatz@cheo.on.ca

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

N/A

Study information

Scientific title

Acronym

In-ex TREND study

Study hypothesis

We 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

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).

Study design

Multi-centre randomised unblinded controlled trial of the mechanical inexsufflator. Randomisation sequence will be stratified by centre with a block-size randomisation protocol.

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Condition

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).

Intervention

1. 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 type

Other

Phase

Not Specified

Drug names

Primary outcome measures

Time 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 measures

1. 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 trial start date

01/10/2007

Overall trial end date

01/04/2009

Reason abandoned

Eligibility

Participant inclusion criteria

1. 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)

Participant type

Patient

Age group

Child

Gender

Both

Target number of participants

128

Participant exclusion criteria

1. 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

Recruitment start date

01/10/2007

Recruitment end date

01/04/2009

Locations

Countries of recruitment

Canada

Trial participating centre

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

Sponsor information

Organisation

Children's Hospital of Eastern Ontario (Canada)

Sponsor details

c/o Sherri Katz
MD
401 Smyth Road
W1406
Ottawa
K1H 8L1
Canada
+1 613 737 7600 ext. 2868
skatz@cheo.on.ca

Sponsor type

Hospital/treatment centre

Website

http://www.cheo.on.ca/english/hub.shtml

Funders

Funder type

Charity

Funder name

Fight Spinal Muscular Atrophy (FightSMA) (USA)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

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

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes