Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy

March 8, 2017 updated by: david orlikowski, Centre d'Investigation Clinique et Technologique 805

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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 in home ventilation unit of Raymond Poincaré hospital.

Selection criteria : Patients with Myotonic dystrophy presenting at least one clinical signs : effort or rest dyspnea, orthopnea, sleepiness, morning headache or VC<50% or Pi max< 60 cm H2O or time of SaO2<90% more than 5 minutes and Hypercapnia > 6.0 kPa.

Study process Preceding screening period within the 3 months before inclusion. Day 1 to day 3 baseline evaluation. Day 3 Inclusion and Randomisation Day 3 to 8 Launch of ventilation Day 8 Home discharge Day 90 Evaluation of efficacy (secondary objectives) and observance.

Duration Participation of a patient 3 months. Period of inclusion 24 months. Total duration of study 30 months.

Study Type

Interventional

Enrollment (Actual)

32

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
    • Paris Area
      • Garches, Paris Area, France, 92380
        • Home ventilation Unit , Raymond Poincaré hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Men or women aged > 18 years
  • Written consent
  • DM1 myotonic dystrophy
  • One clinical signs dyspnea rest/effort, orthopnea, daytime sleepiness (Epworth> 10), morning headache.

Or restrictive syndrome: VC< 50 % or Pi max <60 cm H2O Or time of nocturnal saturation < 90 %, > 5 minutes. AND Hypercapnia > 6.0 kPa Pregnancy test negative or use of contraception for women in age of procreation

Exclusion Criteria:

  • Patient using previously home ventilation within the 6 months before entering the study
  • Patient denying home ventilation
  • Refusal to sign consent
  • impossibility to be followed during 3 months
  • Impossibility to apply ventilation at home
  • No social/health coverage
  • Patient under tutelage

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: SINGLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: AVAPS
Arm assigned to AVAPS mode for nocturnal ventilation with the same setting than bilevel pressure support but with AVAPS mode activated
Device: Nocturnal ventilation
Home ventilation only the mode AVAPS will be used if the patient is randomized in the experimental group.
Other Names:
  • BIPAP A30
ACTIVE_COMPARATOR: Bilevel pressure
Arm treated only with bilevel pressure support for nocturnal ventilation without activation of AVAPS mode
Device: Nocturnal ventilation
Home ventilation only the mode AVAPS will be used if the patient is randomized in the experimental group.
Other Names:
  • BIPAP A30

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
7 days

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Centre d'Investigation Clinique et Technologique 805

Collaborators

Philips Respironics
Adep Assistance

Investigators

  • Principal Investigator: David ORLIKOWSKI, Md-PhD, Raymond Poincare Hospital - Garches - France

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2012

Primary Completion (ACTUAL)

May 1, 2016

Study Completion (ACTUAL)

May 1, 2016

Study Registration Dates

First Submitted

February 8, 2012

First Submitted That Met QC Criteria

February 9, 2012

First Posted (ESTIMATE)

February 10, 2012

Study Record Updates

Last Update Posted (ACTUAL)

March 10, 2017

Last Update Submitted That Met QC Criteria

March 8, 2017

Last Verified

March 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2010-A01306-33

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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