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Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive (DYVINE)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Association Française contre les Myopathies (AFM), Paris
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01225614
First received: October 20, 2010
Last updated: December 18, 2013
Last verified: June 2011
  Purpose

This is a multicenter randomized controlled open labeled study testing efficacy and tolerance of early launching of night non invasive ventilation in patients with myotonic dystrophy type 1(DM1). The object of this project is to estimate the effects of the early introduction of non invasive ventilation on the arisen of complication (non expected hospitalization, tracheostomy even death) with regard to a simple respiratory follow-up in patients affected by myotonic dystrophy.


Condition Intervention Phase
Myopathy
Muscular Weakness
Respiratory Insufficiency
Device: Bilevel pressure ventilator
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Study of Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive Ventilation in Adults With Myotonic Dystrophy Type 1(DM1)

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Rate of patients having a complication (number of non expected hospitalization or death) at 5 years. [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
    Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.


Secondary Outcome Measures:
  • Distribution of survival between the randomisation at 5 years [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
    Distribution of survival between the randomisation at 5 years

  • Number of patients having a formal indication of ventilation [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
    Number of patients having a formal indication of ventilation

  • Number of tracheostomized patients at 5 years [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
    Number of tracheostomized patients at 5 years

  • Number of non expected hospitalizations at 5 years [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
    Number of non expected hospitalizations at 5 years

  • Observance of the ventilation defined by an average minimal use of 4 am by 12:00 pm . [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
    Observance of the ventilation defined by an average minimal use of 4 am by 12:00 pm (determined in a objective way by the counter of the device).

  • Degree of respiratory and sleep impairment at 5 years [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
    Degree of respiratory and sleep impairment at 5 years

  • Quality of life SF36, scales of depression [ Time Frame: 5 YEARS ] [ Designated as safety issue: Yes ]
    Quality of life SF36, scales of depression


Enrollment: 77
Study Start Date: October 2010
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: July 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: bipap ventilation Device: Bilevel pressure ventilator
Nocturnal home ventilation
Other Name: Bilevel pressure ventilator
Active Comparator: Standard care
Standard care and ventilation if occurrence of absolute criteria of ventilation (cf infra).
Device: Bilevel pressure ventilator
Nocturnal home ventilation
Other Name: Bilevel pressure ventilator

Detailed Description:

DM1 is the most frequent genetic myopathy in the adult. Actually there is no curative treatment, and symptomatic cares are essentials. The respiratory impairment is the main cause of morbid-mortality at these patients. The median of survival of the patients affected by DM1 with respiratory failure is of 59 years. Mechanisms of disease are complex implying a central and a direct impairment of respiratory muscles. These patients can present an alveolar hypoventilation, notably during night, not correlated to the muscular weakness. These patients often present a cognitive impairment complicating the interpretation of the clinical symptoms and their compliance to treatments. International recommendations for launching mechanical ventilation in neuromuscular diseases are the presence: 1) at least a clinical sign of alveolar hypoventilation, and one of the following criteria 2) diurnal hypercapnia (> 45 mmHg), 3) restrictive syndrome (vital capacity < 50 % and\or maximal inspiratory pressure < 60 cmH20), 4) the existence of a oxygen night-desaturation (SaO2 < 88 %) of more than 5 minutes. However, a Cochrane meta analyzes underline the absence of randomised study estimating the profit risk in the long term of the night-mechanical ventilation for progressive myopathies such as the DM1. The validity of these criteria and the effect of the ventilation on the survival and the complications were never estimated in DM1. On a retrospective series, the compliance is inferior and the observance is only 20 % a year and the incidence of the complications (death or resort to a tracheostomy) was 6 times as important in non observant patients.

Objective (s) of the clinical study To estimate the efficiency and the tolerance of long term night-non invasive mechanical ventilation in patients affected by DM1.

Main judgment criteria:

Mortality and non programmed hospitalization.

Experimental plan:

Multicenter, national, randomized, controlled, study on 2 parallel groups. The subjects presenting a theoretical indication following consensual criteria of ventilation will be randomized either for a start up of ventilation or for an annual monitoring.

Hypothesis: Early starting of non invasive ventilation allows a reduction of 20 % of the mortality or the number of non-programmed hospitalization compared to the control group for which the rate would be 40 %.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Man or woman of age ≥ 18 years
  • Preliminary medical examination
  • Enlightened and written consent
  • Genetically proved Steinert disease

    1. Presenting at least one of the following 3 criteria

      • A hypercapnia: PaCO2 > 45 mmHg or
      • A night-desaturation: SaO2 < 88 % more than consecutive 5 minutes or
      • Apnea syndrome with significant sleep:index of apnea / hypopnea> a 30 / hour
    2. And with presence of at least a clinical sign: dyspnoea, orthopnea, headaches, asthenia, diurnal sleepiness, or any other sign suggestive of disturbance of the sleep or of respiratory dysfunction

Exclusion Criteria:

  • Age inferior to 18
  • Regime of legal protection
  • Pregnancy
  • Absolute indication for ventilation: clinical signs (dyspnoea, orthopnea, headaches, asthenia, diurnal sleepiness), AND PaCO2 > 60 mmHg, AND night-desaturation < 88 % AND one CV < 50 % of the theoretical or the PIMAX < 60 cm H2O
  • Acute respiratory failure
  • Already ventilated patient
  • Patient under oxygen
  • Not (beneficiary to a regime of Social Security or legal successor)
  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: NCT01225614

Locations
France
Home ventilation unit and intensive care, centre of neuromuscular disease (Garches Mondor Necker Hendaye), Raymond Poincaré hospital Versailles Saint Quentin University.
Garches, France, 92380
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Association Française contre les Myopathies (AFM), Paris
Investigators
Principal Investigator: DAVID ORLIKOWSKI, MD, PhD Assistance Publique - Hôpitaux de Paris
  More Information

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01225614     History of Changes
Other Study ID Numbers: P081221, 2009-A01023-54 (IDRCB)
Study First Received: October 20, 2010
Last Updated: December 18, 2013
Health Authority: France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Home ventilation
Myotonic dystrophy type 1 (DM1)

Additional relevant MeSH terms:
Myotonic Dystrophy
Muscle Weakness
Respiratory Insufficiency
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Muscular Diseases
Muscular Disorders, Atrophic
Muscular Dystrophies
Musculoskeletal Diseases
Myotonic Disorders
Nervous System Diseases
Neurodegenerative Diseases
Neurologic Manifestations
Neuromuscular Diseases
Neuromuscular Manifestations
Pathologic Processes
Respiration Disorders
Respiratory Tract Diseases
Signs and Symptoms

ClinicalTrials.gov processed this record on November 25, 2014