A Phase 3 interventional study of mechanical insufflation - exsufflation and Standard respiratory physiotherapy in Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis and Neuromuscular Diseases, sponsored by Assistance Publique - Hôpitaux de Paris. Terminated at 1 site in France. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2015-05-28.
Sponsored by Assistance Publique - Hôpitaux de Paris · Phase 3, Interventional, and Treatment
The hypothesis is that a mechanical insufflation-exsufflation (MI-E) is associated with a decrease in the number of intubations and more rapid clinical improvement in children and adults with neuromuscular disease who are admitted for an acute respiratory exacerbation.In this prospective, randomised, multicenter study, 55 patients will be treated with standard treatment and a MI-E, and 55 patients with standard treatment and standard respiratory physiotherapy. The primary objective is the reduction of the number of patients requiring invasive ventilatory support (endotracheal intubation or tracheotomy) in the group treated with MI-E (MI-E group). The main secondary objectives are a reduction in hospital stay and an improvement in clinical condition, dyspnea and respiratory muscle function.
Justification Respiratory muscle weakness reduces the efficacy of the cough reflex in patients with neuromuscular disorders and exposes them to the risk of acute respiratory failure. Mechanical insufflation-exsufflation devices assist cough and have been shown to be efficient in increasing the cough expiratory flow in children and adults with neuromuscular disease and decreasing the risk of intubation in a limited population of hospitalized adults with acute respiratory failure.
Primary objective The goal is to record the efficacy of mechanical insufflation-exsufflation (MI-E) during acute respiratory failure in patients with neuromuscular disorders.The primary objective is the reduction of the number of patients requiring invasive ventilatory support (endotracheal intubation or tracheotomy) in the group treated with MI-E (MI-E group) compared to the group treated with traditional chest physiotherapy without MI-E (Control group).
Secondary objectives
In the MI-E group, compared to the Control group:
Type of study Prospective, randomized, controlled, multicenter study
Number of subjects The calculation of the number of subjects is based on two retrospective studies. In the study by VIANELLO, which included 11 adults hospitalized in the ICU for respiratory failure, the number of therapeutic failures, defined as the need for a "mini" tracheotomy or intubation, was significantly less in the group using MI-E than in a group of 16 historical control patients [2 failures in the MI-E group (18%) versus 10 failures in the control group (63%), p\<0.05] (1). Another study reported 19 successes (80%) versus 5 failures on MI-E (2).
480 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 109 are open to participants now.
This study's enrollment of 14 is below the median of 26 across 329 interventional studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,576 studies on the registry; 1,028 are open to participants now.
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Pediatric or adult patients with chronic neuromuscular disorders, such as spinal muscular atrophy, Duchenne muscular dystrophy, other congenital myopathy, or amyotrophic lateral sclerosis (ALS), hospitalized for acute respiratory failure, as defined by:
Exclusion Criteria:
patients treated with standard treatment and a mechanical insufflation-exsufflation
Device: mechanical insufflation - exsufflation
Patients with standard treatment and standard respiratory physiotherapy
Device: Standard respiratory physiotherapy
Patients will receive MI-E treatment with the following settings: insufflation pressure of at least +30 cm H2O and an exsufflation pressure ≥ -30 cm H2O. There will be at least 6 hyperinflation/exsufflation sequences per session of chest physiotherapy. There will be at least two daily sessions done routinely by the respiratory therapist at 8 hour intervals.
Also known as: Mechanical insufflation-exsufflation devices assist cough
Traditional chest physiotherapy without mechanical insufflation-exsufflation
Reduction of the number of patients requiring invasive ventilatory support in the group treated with MI-E (MI-E group) compared to the group treated with traditional chest physiotherapy without MI-E (Control group).
Time frame: During the treatment phase
Decrease in the length of hospitalization in the intensive care unit (ICU) (if necessary)
Time frame: During the treatment phase
Decrease in the total length of hospitalization
Time frame: During the treatment phase
Decrease in the incidence of bronchoscopy-assisted aspiration
Time frame: During the treatment phase
Decrease in the duration of oxygen therapy
Time frame: During the treatment phase
Decrease in the daily length of noninvasive positive pressure ventilation (NPPV)
Time frame: During the treatment phase
Improvement in blood gases on room air during hospitalization and improvement of the peak cough flow (PCF)
Time frame: During the treatment phase
Improvement of the vital capacity (VC), maximal inspiratory (PImax) and expiratory (PEmax) pressures, sniff nasal inspiratory pressure (SNIP), peak expiratory flow (PEF) and dyspnea during hospitalization
Time frame: During the treatment phase
Decrease in the number of secondary tracheotomies (for weaning of ventilatory support)
Time frame: During the treatment phase
The record gives a start of Jun 2009, a primary completion of Feb 2011 and a completion of Dec 2011. It does not say whether these dates are actual or estimated.
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This study is terminated, as verified in May 2015. You cannot join it, but the record below documents what was studied.
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