Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Centre d'Investigation Clinique et Technologique 805
Sponsor:
Collaborators:
Adep Assistance
Philips Respironics
Information provided by (Responsible Party):
david orlikowski, Centre d'Investigation Clinique et Technologique 805
ClinicalTrials.gov Identifier:
NCT01530841
First received: February 8, 2012
Last updated: July 24, 2014
Last verified: July 2014
  Purpose

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 Intervention Phase
Myopathy
Device: Nocturnal ventilation
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
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

Resource links provided by NLM:


Further study details as provided by Centre d'Investigation Clinique et Technologique 805:

Primary Outcome Measures:
  • arterial PCO2 under ventilation [ Time Frame: 7 days ] [ Designated as safety issue: No ]
    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. [ Time Frame: 90 days ] [ Designated as safety issue: No ]
    To evaluate efficacy of AVAPS Mode at day 90 on daytime arterial PCO2 after launching ventilation.

  • Compliance to ventilation [ Time Frame: 7 and 90 days ] [ Designated as safety issue: No ]
    To evaluate compliance (h/24h) to ventilation at days 7 and 90.

  • Symptoms [ Time Frame: 90 days ] [ Designated as safety issue: No ]
    To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90.

  • Sleep studies [ Time Frame: 90 days ] [ Designated as safety issue: No ]
    To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2 at day 90.

  • OBJECTIVE SLEEPINESS [ Time Frame: 90 days ] [ Designated as safety issue: Yes ]
    To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90.

  • Respiratory parameters [ Time Frame: 90 days ] [ Designated as safety issue: No ]
    To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures.


Estimated Enrollment: 32
Study Start Date: February 2011
Estimated Study Completion Date: June 2015
Estimated Primary Completion Date: April 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01530841

Contacts
Contact: David Orlikowski, MD-PhD 33-1-47-10-77-76 david.orlikowski@rpc.aphp.fr
Contact: Frédéric Lofaso, MD-PhD 33-1-47-10-79-41 f.lofaso@rpc.aphp.fr

Locations
France
Home ventilation Unit , Raymond Poincaré hospital Recruiting
Garches, Paris Area, France, 92380
Contact: David Orlikowski, MD, PhD    33147107776    david.orlikowski@rpc.aphp.fr   
Contact: Sandra Pottier, CRA    33 1 47 10 44 69    sandra.pottier@rpc.aphp.fr   
Sub-Investigator: Helene Prigent, MD, PhD         
Principal Investigator: David Orlikowski, MD, PhD         
Sub-Investigator: Mourad Machou, MD         
Sub-Investigator: Maria Antonia Quera Salva, MD, PhD         
Sub-Investigator: Frederic Lofaso, MD, PhD         
Sub-Investigator: Julie Nardi, MD         
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

No publications provided

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
Study First Received: February 8, 2012
Last Updated: July 24, 2014
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Centre d'Investigation Clinique et Technologique 805:
Myotonic dystrophy type 1
Home ventilation
Respiratory insufficiency
Neuromuscular

Additional relevant MeSH terms:
Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on September 18, 2014