Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders (Nemucough)

This study has been terminated.
(No inclusion)
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT00839033
First received: February 6, 2009
Last updated: December 26, 2011
Last verified: August 2011
  Purpose

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.


Condition Intervention Phase
Duchenne Muscular Dystrophy
Amyotrophic Lateral Sclerosis
Neuromuscular Diseases
Device: mechanical insufflation - exsufflation
Device: Standard respiratory physiotherapy
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evaluation of a Mechanical Insufflation-exsufflation Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders: a Prospective, Randomized, Controlled, Multicenter Study

Resource links provided by NLM:


Further study details as provided by Assistance Publique - Hôpitaux de Paris:

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). [ Time Frame: During the treatment phase ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Decrease in the length of hospitalization in the intensive care unit (ICU) (if necessary) [ Time Frame: During the treatment phase ] [ Designated as safety issue: No ]
  • Decrease in the total length of hospitalization [ Time Frame: During the treatment phase ] [ Designated as safety issue: No ]
  • Decrease in the incidence of bronchoscopy-assisted aspiration [ Time Frame: During the treatment phase ] [ Designated as safety issue: No ]
  • Decrease in the duration of oxygen therapy [ Time Frame: During the treatment phase ] [ Designated as safety issue: No ]
  • Decrease in the daily length of noninvasive positive pressure ventilation (NPPV) [ Time Frame: During the treatment phase ] [ Designated as safety issue: No ]
  • Improvement in blood gases on room air during hospitalization and improvement of the peak cough flow (PCF) [ Time Frame: During the treatment phase ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Decrease in the number of secondary tracheotomies (for weaning of ventilatory support) [ Time Frame: During the treatment phase ] [ Designated as safety issue: No ]

Enrollment: 14
Study Start Date: June 2009
Estimated Study Completion Date: December 2011
Primary Completion Date: February 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
patients treated with standard treatment and a mechanical insufflation-exsufflation
Device: mechanical insufflation - exsufflation
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.
Other Name: Mechanical insufflation-exsufflation devices assist cough
Active Comparator: 2
Patients with standard treatment and standard respiratory physiotherapy
Device: Standard respiratory physiotherapy
Traditional chest physiotherapy without mechanical insufflation-exsufflation
Other Name: Standard respiratory physiotherapy

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:

  1. Decrease in the length of hospitalization in the intensive care unit (ICU)
  2. Decrease in the total length of hospitalization
  3. Decrease in the incidence of bronchoscopy-assisted aspiration
  4. Decrease in the duration of oxygen therapy
  5. Decrease in the daily length of noninvasive positive pressure ventilation (NPPV)
  6. Improvement in blood gases on room air during hospitalization
  7. Improvement of the peak cough flow (PCF)
  8. 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.
  9. 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).

  Eligibility

Ages Eligible for Study:   4 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00839033

Locations
France
Hospital Armand Trousseau, Pediatric Pulmonology Department and INSERM UMR S-893
Paris, France, 75012
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Brigitte FAUROUX, MD PhD Assistance Publique - Hôpitaux de Paris
  More Information

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT00839033     History of Changes
Other Study ID Numbers: P080406
Study First Received: February 6, 2009
Last Updated: December 26, 2011
Health Authority: France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Cough assisted device
Neuromuscular disease
Respiratory exacerbation
Invasive ventilation,
Chest physiotherapy
Congenital myopathies

Additional relevant MeSH terms:
Muscular Disorders, Atrophic
Muscular Diseases
Amyotrophic Lateral Sclerosis
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Neuromuscular Diseases
Sclerosis
Motor Neuron Disease
Respiratory Distress Syndrome, Adult
Respiratory Insufficiency
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Musculoskeletal Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Pathologic Processes
Lung Diseases
Respiratory Tract Diseases
Respiration Disorders

ClinicalTrials.gov processed this record on July 24, 2014