search
Back to results

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
Centre d'Investigation Clinique et Technologique 805
About
Eligibility
Locations
Arms
Outcomes
Full info

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

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

First Posted
February 8, 2012
Last Updated
March 8, 2017
Sponsor
Centre d'Investigation Clinique et Technologique 805
Collaborators
Adep Assistance, Philips Respironics
search

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

We'll reach out to this number within 24 hrs