Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy

Myopathy Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01530841
• Other study ID #: 2010-A01306-33
• Status: Completed
• Phase: N/A
• First received: February 8, 2012
• Last updated: March 8, 2017
• Start date: July 2012
• Est. completion date: May 2016

Study information

• Verified date: March 2017
• Source: Centre d'Investigation Clinique et Technologique 805
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: May 2016
• Est. primary completion date: May 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Myopathy
• Myotonic Dystrophy

Intervention

Device:
• Nocturnal ventilation
Home ventilation only the mode AVAPS will be used if the patient is randomized in the experimental group.

Locations

Country	Name	City	State
France	Home ventilation Unit , Raymond Poincaré hospital	Garches	Paris Area

Sponsors (3)

Lead Sponsor	Collaborator
Centre d'Investigation Clinique et Technologique 805	Adep Assistance, Philips Respironics

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	7 days
Secondary	daytime arterial PCO2 after launching ventilation.	To evaluate efficacy of AVAPS Mode at day 90 on daytime arterial PCO2 after launching ventilation.	90 days
Secondary	Compliance to ventilation	To evaluate compliance (h/24h) to ventilation at days 7 and 90.	7 and 90 days
Secondary	Symptoms	To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90.	90 days
Secondary	Sleep studies	To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2 at day 90.	90 days
Secondary	OBJECTIVE SLEEPINESS	To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90.	90 days
Secondary	Respiratory parameters	To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures.	90 days