NCT01530841

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
32

participants targeted

Target at P25-P50 for not_applicable

Timeline
Completed

Started Jul 2012

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 8, 2012

Completed
2 days until next milestone

First Posted

Study publicly available on registry

February 10, 2012

Completed
5 months until next milestone

Study Start

First participant enrolled

July 1, 2012

Completed
3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2016

Completed
Last Updated

March 10, 2017

Status Verified

March 1, 2017

Enrollment Period

3.8 years

First QC Date

February 8, 2012

Last Update Submit

March 8, 2017

Conditions

Myopathy

Keywords

Myotonic dystrophy type 1Home ventilationRespiratory insufficiencyNeuromuscular

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (6)

  • daytime arterial PCO2 after launching ventilation.

    90 days

  • Compliance to ventilation

    7 and 90 days

  • Symptoms

    90 days

  • Sleep studies

    90 days

  • OBJECTIVE SLEEPINESS

    90 days

  • +1 more secondary outcomes

Study Arms (2)

AVAPS

EXPERIMENTAL

Arm assigned to AVAPS mode for nocturnal ventilation with the same setting than bilevel pressure support but with AVAPS mode activated

Device: Nocturnal ventilation

Bilevel pressure

ACTIVE COMPARATOR

Arm treated only with bilevel pressure support for nocturnal ventilation without activation of AVAPS mode

Device: Nocturnal ventilation

Interventions

Nocturnal ventilationDEVICE

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

Also known as: BIPAP A30
AVAPSBilevel pressure

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Home ventilation Unit , Raymond Poincaré hospital

Garches, Paris Area, 92380, France

Location

MeSH Terms

Conditions

Muscular DiseasesMyotonic DystrophyRespiratory Insufficiency

Condition Hierarchy (Ancestors)

Musculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesMuscular DystrophiesMuscular Disorders, AtrophicMyotonic DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesRespiration DisordersRespiratory Tract Diseases

Study Officials

  • David ORLIKOWSKI, Md-PhD

    Raymond Poincare Hospital - Garches - France

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor, MD, PhD

Study Record Dates

First Submitted

February 8, 2012

First Posted

February 10, 2012

Study Start

July 1, 2012

Primary Completion

May 1, 2016

Study Completion

May 1, 2016

Last Updated

March 10, 2017

Record last verified: 2017-03

Locations

FR(1)