Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy

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

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.

Condition or disease	Intervention/treatment	Phase
Myopathy	Device: Nocturnal ventilation	Not Applicable

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 32 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Single (Participant)
• Primary Purpose: Treatment
• 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 Start Date: July 2012
• Actual Primary Completion Date: May 2016
• Actual Study Completion Date: May 2016

Arms and Interventions

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	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
Active Comparator: Bilevel pressure; Arm treated only with bilevel pressure support for nocturnal ventilation without activation of AVAPS mode	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

Outcome Measures

Primary Outcome Measures :

1. 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

Secondary Outcome Measures :

1. 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.
2. Compliance to ventilation [ Time Frame: 7 and 90 days ]
   To evaluate compliance (h/24h) to ventilation at days 7 and 90.
3. Symptoms [ Time Frame: 90 days ]
   To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90.
4. Sleep studies [ Time Frame: 90 days ]
   To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2 at day 90.
5. OBJECTIVE SLEEPINESS [ Time Frame: 90 days ]
   To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90.
6. Respiratory parameters [ Time Frame: 90 days ]
   To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

France

Home ventilation Unit , Raymond Poincaré hospital

Garches, Paris Area, France, 92380

Sponsors and Collaborators

Centre d'Investigation Clinique et Technologique 805

Adep Assistance

Philips Respironics

Investigators

• Principal Investigator: David ORLIKOWSKI, Md-PhD; Raymond Poincare Hospital - Garches - France

More Information

• Responsible Party: david orlikowski, Professor, MD, PhD, Centre d'Investigation Clinique et Technologique 805
• ClinicalTrials.gov Identifier: NCT01530841 History of Changes
• Other Study ID Numbers: 2010-A01306-33
• First Posted: February 10, 2012
• Last Update Posted: March 10, 2017
• Last Verified: March 2017

Keywords provided by david orlikowski, Centre d'Investigation Clinique et Technologique 805:

Myotonic dystrophy type 1; Home ventilation; Respiratory insufficiency; Neuromuscular

Additional relevant MeSH terms:

Myotonic Dystrophy; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Muscular Dystrophies; Muscular Disorders, Atrophic; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn