Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy
Primary Purpose
Myopathy
Status
Completed
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Nocturnal ventilation
Sponsored by
About this trial
This is an interventional treatment trial for Myopathy focused on measuring Myotonic dystrophy type 1, Home ventilation, Respiratory insufficiency, Neuromuscular
Eligibility Criteria
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
Sites / Locations
- Home ventilation Unit , Raymond Poincaré hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
AVAPS
Bilevel pressure
Arm Description
Arm assigned to AVAPS mode for nocturnal ventilation with the same setting than bilevel pressure support but with AVAPS mode activated
Arm treated only with bilevel pressure support for nocturnal ventilation without activation of AVAPS mode
Outcomes
Primary Outcome Measures
arterial PCO2 under ventilation
To evaluate efficacy of AVAPS Mode versus bilevel pressure mode at day 7 on arterial PCO2 under ventilation after launching ventilation
Secondary Outcome Measures
daytime arterial PCO2 after launching ventilation.
To evaluate efficacy of AVAPS Mode at day 90 on daytime arterial PCO2 after launching ventilation.
Compliance to ventilation
To evaluate compliance (h/24h) to ventilation at days 7 and 90.
Symptoms
To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90.
Sleep studies
To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2 at day 90.
OBJECTIVE SLEEPINESS
To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90.
Respiratory parameters
To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures.
Full Information
NCT ID
NCT01530841
First Posted
February 8, 2012
Last Updated
March 8, 2017
Sponsor
Centre d'Investigation Clinique et Technologique 805
Collaborators
Adep Assistance, Philips Respironics
1. Study Identification
Unique Protocol Identification Number
NCT01530841
Brief Title
Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy
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
Study Type
Interventional
2. Study Status
Record Verification Date
March 2017
Overall Recruitment Status
Completed
Study Start Date
July 2012 (undefined)
Primary Completion Date
May 2016 (Actual)
Study Completion Date
May 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Centre d'Investigation Clinique et Technologique 805
Collaborators
Adep Assistance, Philips Respironics
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myopathy
Keywords
Myotonic dystrophy type 1, Home ventilation, Respiratory insufficiency, Neuromuscular
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
Participant
Allocation
Randomized
Enrollment
32 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AVAPS
Arm Type
Experimental
Arm Description
Arm assigned to AVAPS mode for nocturnal ventilation with the same setting than bilevel pressure support but with AVAPS mode activated
Arm Title
Bilevel pressure
Arm Type
Active Comparator
Arm Description
Arm treated only with bilevel pressure support for nocturnal ventilation without activation of AVAPS mode
Intervention Type
Device
Intervention Name(s)
Nocturnal ventilation
Other Intervention Name(s)
BIPAP A30
Intervention Description
Home ventilation only the mode AVAPS will be used if the patient is randomized in the experimental group.
Primary Outcome Measure Information:
Title
arterial PCO2 under ventilation
Description
To evaluate efficacy of AVAPS Mode versus bilevel pressure mode at day 7 on arterial PCO2 under ventilation after launching ventilation
Time Frame
7 days
Secondary Outcome Measure Information:
Title
daytime arterial PCO2 after launching ventilation.
Description
To evaluate efficacy of AVAPS Mode at day 90 on daytime arterial PCO2 after launching ventilation.
Time Frame
90 days
Title
Compliance to ventilation
Description
To evaluate compliance (h/24h) to ventilation at days 7 and 90.
Time Frame
7 and 90 days
Title
Symptoms
Description
To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90.
Time Frame
90 days
Title
Sleep studies
Description
To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2 at day 90.
Time Frame
90 days
Title
OBJECTIVE SLEEPINESS
Description
To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90.
Time Frame
90 days
Title
Respiratory parameters
Description
To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures.
Time Frame
90 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David ORLIKOWSKI, Md-PhD
Organizational Affiliation
Raymond Poincare Hospital - Garches - France
Official's Role
Principal Investigator
Facility Information:
Facility Name
Home ventilation Unit , Raymond Poincaré hospital
City
Garches
State/Province
Paris Area
ZIP/Postal Code
92380
Country
France
12. IPD Sharing Statement
Learn more about this trial
Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy
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