Clinical Trial: Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Prospective Randomized Controlled Single Blind Study of Efficacy and Tolerance of AVAPS Mode Compared to Bilevel Pressure Ventilation un Adult Patients With Myotonic Dystrophy

Brief Summary: Myotonic dystrophy type 1 is a myopathy with complex respiratory pattern and at risk to develop respiratory failure. Classical mode of ventilation are sometimes not tolerated or ineffective in this population. New modes of nocturnal ventilation by combining both volumetric and barometric advantages. The aim of this study is to compare effect of AVAPS mode to bilevel pressure support.

Detailed Summary:

Justification of study

Respiratory abnormalities are complex in Myotonic dystrophy type 1. Some patients presented with isolated alveolar hypoventilation and breathe rhythm irregularity. Nocturnal ventilation is usually proposed but usual modes of ventilation can't provide enough respiratory assistance for patients especially during REM sleep or too much respiratory assistance increasing the risk of asynchrony. The goal of this study is to evaluate the effect of the mode AVAPS (a mode permitting a pressure support with guaranteed volume and offering advantage of volume and pressure support with Bipap A30 Phillips Respironics) compared to bilevel pressure support.

Main Objective To evaluate efficacy of AVAPS Mode at day 7 on arterial PCO2 under ventilation after launching ventilation.

Secondary Objectives To evaluate efficacy of AVAPS Mode at day 90 on daytime arterial PCO2 after launching ventilation.

To evaluate compliance to ventilation at day 7 and 90. To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90.

To evaluate quality of life at day 1 and 90. To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2.

To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90.

To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures.

Type of study: Prospective, monocentre, randomized, controlled single blind study on 2 parallel group.

Number of subjects: 32 patients recruited
Sponsor: Centre d'Investigation Clinique et Technologique 805

Current Primary Outcome: arterial PCO2 under ventilation [ Time Frame: 7 days ]

To evaluate efficacy of AVAPS Mode versus bilevel pressure mode at day 7 on arterial PCO2 under ventilation after launching ventilation


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • daytime arterial PCO2 after launching ventilation. [ Time Frame: 90 days ]
    To evaluate efficacy of AVAPS Mode at day 90 on daytime arterial PCO2 after launching ventilation.
  • Compliance to ventilation [ Time Frame: 7 and 90 days ]
    To evaluate compliance (h/24h) to ventilation at days 7 and 90.
  • Symptoms [ Time Frame: 90 days ]
    To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90.
  • Sleep studies [ Time Frame: 90 days ]
    To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2 at day 90.
  • OBJECTIVE SLEEPINESS [ Time Frame: 90 days ]
    To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90.
  • Respiratory parameters [ Time Frame: 90 days ]
    To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures.


Original Secondary Outcome: Same as current

Information By: Centre d'Investigation Clinique et Technologique 805

Dates:
Date Received: February 8, 2012
Date Started: July 2012
Date Completion:
Last Updated: March 8, 2017
Last Verified: March 2017