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Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy

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ClinicalTrials.gov Identifier: NCT01530841
Recruitment Status : Completed
First Posted : February 10, 2012
Last Update Posted : March 10, 2017
Sponsor:
Collaborators:
Adep Assistance
Philips Respironics
Information provided by (Responsible Party):
david orlikowski, Centre d'Investigation Clinique et Technologique 805

Tracking Information
First Submitted Date  ICMJE February 8, 2012
First Posted Date  ICMJE February 10, 2012
Last Update Posted Date March 10, 2017
Study Start Date  ICMJE July 2012
Actual Primary Completion Date May 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 9, 2012)
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 Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01530841 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: February 9, 2012)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy
Official Title  ICMJE 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 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  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Condition  ICMJE Myopathy
Intervention  ICMJE Device: Nocturnal ventilation
Home ventilation only the mode AVAPS will be used if the patient is randomized in the experimental group.
Other Name: BIPAP A30
Study Arms  ICMJE
  • Experimental: AVAPS
    Arm assigned to AVAPS mode for nocturnal ventilation with the same setting than bilevel pressure support but with AVAPS mode activated
    Intervention: Device: Nocturnal ventilation
  • Active Comparator: Bilevel pressure
    Arm treated only with bilevel pressure support for nocturnal ventilation without activation of AVAPS mode
    Intervention: Device: Nocturnal ventilation
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 9, 2012)
32
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE May 2016
Actual Primary Completion Date May 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01530841
Other Study ID Numbers  ICMJE 2010-A01306-33
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party david orlikowski, Centre d'Investigation Clinique et Technologique 805
Study Sponsor  ICMJE Centre d'Investigation Clinique et Technologique 805
Collaborators  ICMJE
  • Adep Assistance
  • Philips Respironics
Investigators  ICMJE
Principal Investigator: David ORLIKOWSKI, Md-PhD Raymond Poincare Hospital - Garches - France
PRS Account Centre d'Investigation Clinique et Technologique 805
Verification Date March 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP