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

NCT00839033 | Terminated Phase 3

• 1 other identifier: P080406
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy; Amyotrophic Lateral Sclerosis; Neuromuscular Diseases

Keywords

Cough assisted device; Neuromuscular disease; Respiratory exacerbation; Invasive ventilation,; Chest physiotherapy; Congenital myopathies

Outcome Measures

Primary Outcomes (1)

• 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).

  During the treatment phase

Secondary Outcomes (8)

• Decrease in the length of hospitalization in the intensive care unit (ICU) (if necessary)

  During the treatment phase

• Decrease in the total length of hospitalization

  During the treatment phase

• Decrease in the incidence of bronchoscopy-assisted aspiration

  During the treatment phase

• Decrease in the duration of oxygen therapy

  During the treatment phase

• Decrease in the daily length of noninvasive positive pressure ventilation (NPPV)

  During the treatment phase

Study Arms (2)

1

EXPERIMENTAL

patients treated with standard treatment and a mechanical insufflation-exsufflation

Device: mechanical insufflation - exsufflation

2

ACTIVE COMPARATOR

Patients with standard treatment and standard respiratory physiotherapy

Device: Standard respiratory physiotherapy

Interventions

mechanical insufflation - exsufflation DEVICE

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

1

Standard respiratory physiotherapy DEVICE

Traditional chest physiotherapy without mechanical insufflation-exsufflation

2

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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%.

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Hospital Armand Trousseau, Pediatric Pulmonology Department and INSERM UMR S-893

Paris, 75012, France

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Amyotrophic Lateral Sclerosis; Neuromuscular Diseases; Myotonia Congenita

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Spinal Cord Diseases; Central Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System

Study Officials

• Brigitte FAUROUX, MD PhD

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 6, 2009
• First Posted: February 9, 2009
• Study Start: June 1, 2009
• Primary Completion: February 1, 2011
• Study Completion: December 1, 2011
• Last Updated: May 28, 2015

Record last verified: 2015-05

Locations

FR (1)