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Duchenne Muscular DystrophyPhase 3NemucoughNCT00839033

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

A Phase 3 study testing mechanical insufflation - exsufflation and Standard respiratory physiotherapy in people with Duchenne Muscular Dystrophy and Amyotrophic Lateral Sclerosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 4 years and older
What is being tested?
mechanical insufflation - exsufflation and Standard respiratory physiotherapy
Type of treatment
Medical device
Could I receive a placebo?
No, everyone receives an active treatment
How many people
14 participants
Where
1 siteFrance
Study dates
Jun 2009to Dec 2011
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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%.

Who cannot join

  • 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
Show all 13Show fewer
  • 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

Trial locations and contacts

1 site in 1 country.

France1 site

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

    Paris, 75012

Other trials in France

About this study

Official title: Evaluation of a Mechanical Insufflation-exsufflation Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders: a Prospective, Randomized, Controlled, Multicenter Study

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.

Read the 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).

Being tested
mechanical insufflation - exsufflation, Standard respiratory physiotherapy
Keywords
Cough assisted device, Neuromuscular disease, Respiratory exacerbation, Invasive ventilation,, Chest physiotherapy, Congenital myopathies
Medical subject headings
Muscular Dystrophy, Duchenne, Amyotrophic Lateral Sclerosis, Neuromuscular Diseases, Myotonia Congenita

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • mechanical insufflation - exsufflationDevice

    Also called Mechanical insufflation-exsufflation devices assist cough

    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.

  • Standard respiratory physiotherapyDevice

    Traditional chest physiotherapy without mechanical insufflation-exsufflation

Study groups in NCT00839033
Study groupRoleWhat they receive
1Experimentalpatients treated with standard treatment and a mechanical insufflation-exsufflation
Receives: Device: mechanical insufflation - exsufflation
2Active comparatorPatients with standard treatment and standard respiratory physiotherapy
Receives: Device: Standard respiratory physiotherapy

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: During the treatment phase

Secondary outcomes (8)

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

    Measured: During the treatment phase

  2. Decrease in the total length of hospitalization

    Measured: During the treatment phase

  3. Decrease in the incidence of bronchoscopy-assisted aspiration

    Measured: During the treatment phase

  4. Decrease in the duration of oxygen therapy

    Measured: During the treatment phase

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

    Measured: During the treatment phase

  6. Improvement in blood gases on room air during hospitalization and improvement of the peak cough flow (PCF)

    Measured: During the treatment phase

  7. 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

    Measured: During the treatment phase

  8. Decrease in the number of secondary tracheotomies (for weaning of ventilatory support)

    Measured: During the treatment phase

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Feb 2009 (estimated)
  2. Study startJun 2009
  3. Primary completionFeb 2011
  4. Study completionDec 2011

Why it stopped: No inclusion

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
14 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00839033
Sponsor's protocol ID
P080406
Acronym
Nemucough
Status last verified
May 2015
First submitted
6 Feb 2009
Last updated
28 May 2015
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing mechanical insufflation - exsufflation and Standard respiratory physiotherapy in people with Duchenne Muscular Dystrophy and Amyotrophic Lateral Sclerosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 28 May 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: No inclusion). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00839033.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2015.

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