Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy
Keywords
Abstract
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.
Dates
| Last Verified: | 02/28/2017 |
| First Submitted: | 02/07/2012 |
| Estimated Enrollment Submitted: | 02/08/2012 |
| First Posted: | 02/09/2012 |
| Last Update Submitted: | 03/07/2017 |
| Last Update Posted: | 03/09/2017 |
| Actual Study Start Date: | 06/30/2012 |
| Estimated Primary Completion Date: | 04/30/2016 |
| Estimated Study Completion Date: | 04/30/2016 |
Condition or disease
Intervention/treatment
Device: Nocturnal ventilation
Phase
Arm Groups
| 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 | |
| Active Comparator: Bilevel pressure Arm treated only with bilevel pressure support for nocturnal ventilation without activation of AVAPS mode |
Eligibility Criteria
| Ages Eligible for Study | 18 Years To 18 Years |
| Sexes Eligible for Study | All |
| Accepts Healthy Volunteers | Yes |
| 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 |
Outcome
Primary Outcome Measures
1. arterial PCO2 under ventilation [7 days]
Secondary Outcome Measures
1. daytime arterial PCO2 after launching ventilation. [90 days]
2. Compliance to ventilation [7 and 90 days]
3. Symptoms [90 days]
4. Sleep studies [90 days]
5. OBJECTIVE SLEEPINESS [90 days]
6. Respiratory parameters [90 days]
