Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders (Nemucough)
Primary Purpose
Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis, Neuromuscular Diseases
Status
Terminated
Phase
Phase 3
Locations
France
Study Type
Interventional
Intervention
mechanical insufflation - exsufflation
Standard respiratory physiotherapy
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring Cough assisted device, Neuromuscular disease, Respiratory exacerbation, Invasive ventilation,, Chest physiotherapy, Congenital myopathies
Eligibility Criteria
Inclusion Criteria:
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:
- Persistent bronchial encumbrance (> 2 days) despite regular treatment in the homecare setting, associated with-Oxygen desaturation on room air, defined by a pulse oximetry (SaO2) <95%) or
- In patients not receiving long-term NPPV: the need to institute NPPV-In patients receiving long-term NPPV: the need to increase the daily length of NPPV by at least 25%.
Exclusion Criteria:
- Need for immediate intubation (alteration in consciousness, coma, hemodynamic disorders)
- Multiple organ failure (e.g., associated cardiac failure)
- In adults: respiratory rate >30/min, pH < 7.35, PaCO2 > 50 mm Hg
- Facial deformity or anomaly which prevents the use of a mouthpiece or mask
- Patients who signed a refusal to be intubated regardless of the progression of their disease
- Patients on long-term oxygen therapy
- Tracheotomized patients
- Patients requiring the use of an intrapulmonary percussive ventilation device during hospitalization
- Acute neuromuscular disorder of known or unknown etiology
- Associated lung disease such as chronic obstructive pulmonary disease (COPD)
- Refusal of patient consent and/or parental consent in the case of a minor
- Uncooperative patients
- Patients < 4 years old
Sites / Locations
- Hospital Armand Trousseau, Pediatric Pulmonology Department and INSERM UMR S-893
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
1
2
Arm Description
patients treated with standard treatment and a mechanical insufflation-exsufflation
Patients with standard treatment and standard respiratory physiotherapy
Outcomes
Primary Outcome Measures
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).
Secondary Outcome Measures
Decrease in the length of hospitalization in the intensive care unit (ICU) (if necessary)
Decrease in the total length of hospitalization
Decrease in the incidence of bronchoscopy-assisted aspiration
Decrease in the duration of oxygen therapy
Decrease in the daily length of noninvasive positive pressure ventilation (NPPV)
Improvement in blood gases on room air during hospitalization and improvement of the peak cough flow (PCF)
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
Decrease in the number of secondary tracheotomies (for weaning of ventilatory support)
Full Information
NCT ID
NCT00839033
First Posted
February 6, 2009
Last Updated
May 27, 2015
Sponsor
Assistance Publique - Hôpitaux de Paris
1. Study Identification
Unique Protocol Identification Number
NCT00839033
Brief Title
Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
Acronym
Nemucough
Official Title
Evaluation of a Mechanical Insufflation-exsufflation Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders: a Prospective, Randomized, Controlled, Multicenter Study
Study Type
Interventional
2. Study Status
Record Verification Date
May 2015
Overall Recruitment Status
Terminated
Why Stopped
No inclusion
Study Start Date
June 2009 (undefined)
Primary Completion Date
February 2011 (Actual)
Study Completion Date
December 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
Detailed Description
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:
Decrease in the length of hospitalization in the intensive care unit (ICU)
Decrease in the total length of hospitalization
Decrease in the incidence of bronchoscopy-assisted aspiration
Decrease in the duration of oxygen therapy
Decrease in the daily length of noninvasive positive pressure ventilation (NPPV)
Improvement in blood gases on room air during hospitalization
Improvement of the peak cough flow (PCF)
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.
Decrease in the number of secondary tracheotomies (for weaning of ventilatory support)
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis, Neuromuscular Diseases
Keywords
Cough assisted device, Neuromuscular disease, Respiratory exacerbation, Invasive ventilation,, Chest physiotherapy, Congenital myopathies
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
14 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Experimental
Arm Description
patients treated with standard treatment and a mechanical insufflation-exsufflation
Arm Title
2
Arm Type
Active Comparator
Arm Description
Patients with standard treatment and standard respiratory physiotherapy
Intervention Type
Device
Intervention Name(s)
mechanical insufflation - exsufflation
Other Intervention Name(s)
Mechanical insufflation-exsufflation devices assist cough
Intervention Description
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.
Intervention Type
Device
Intervention Name(s)
Standard respiratory physiotherapy
Intervention Description
Traditional chest physiotherapy without mechanical insufflation-exsufflation
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
Decrease in the length of hospitalization in the intensive care unit (ICU) (if necessary)
Time Frame
During the treatment phase
Title
Decrease in the total length of hospitalization
Time Frame
During the treatment phase
Title
Decrease in the incidence of bronchoscopy-assisted aspiration
Time Frame
During the treatment phase
Title
Decrease in the duration of oxygen therapy
Time Frame
During the treatment phase
Title
Decrease in the daily length of noninvasive positive pressure ventilation (NPPV)
Time Frame
During the treatment phase
Title
Improvement in blood gases on room air during hospitalization and improvement of the peak cough flow (PCF)
Time Frame
During the treatment phase
Title
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
Title
Decrease in the number of secondary tracheotomies (for weaning of ventilatory support)
Time Frame
During the treatment phase
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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:
Persistent bronchial encumbrance (> 2 days) despite regular treatment in the homecare setting, associated with-Oxygen desaturation on room air, defined by a pulse oximetry (SaO2) <95%) or
In patients not receiving long-term NPPV: the need to institute NPPV-In patients receiving long-term NPPV: the need to increase the daily length of NPPV by at least 25%.
Exclusion Criteria:
Need for immediate intubation (alteration in consciousness, coma, hemodynamic disorders)
Multiple organ failure (e.g., associated cardiac failure)
In adults: respiratory rate >30/min, pH < 7.35, PaCO2 > 50 mm Hg
Facial deformity or anomaly which prevents the use of a mouthpiece or mask
Patients who signed a refusal to be intubated regardless of the progression of their disease
Patients on long-term oxygen therapy
Tracheotomized patients
Patients requiring the use of an intrapulmonary percussive ventilation device during hospitalization
Acute neuromuscular disorder of known or unknown etiology
Associated lung disease such as chronic obstructive pulmonary disease (COPD)
Refusal of patient consent and/or parental consent in the case of a minor
Uncooperative patients
Patients < 4 years old
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Brigitte FAUROUX, MD PhD
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hospital Armand Trousseau, Pediatric Pulmonology Department and INSERM UMR S-893
City
Paris
ZIP/Postal Code
75012
Country
France
12. IPD Sharing Statement
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Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
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