MyopathyNCT01530841
Efficacy and Tolerance of AVAPS Mode in Myotonic Dystrophy
A study testing Nocturnal ventilation in people with Myopathy, run by Centre d'Investigation Clinique et Technologique 805 at 1 site in France.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myopathy
- What is being tested?
- Nocturnal ventilation
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 1 siteFrance
- Study dates
- Jul 2012to May 2016
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
France1 site
Home ventilation Unit , Raymond Poincaré hospital
Garches, Paris Area, 92380
About this study
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
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.
Read the 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.
- Conditions
- Myopathy
- Being tested
- Nocturnal ventilation
- Keywords
- Myotonic dystrophy type 1, Home ventilation, Respiratory insufficiency, Neuromuscular
- Medical subject headings
- Muscular Diseases, Myotonic Dystrophy, Respiratory Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nocturnal ventilationDevice
Also called BIPAP A30
Home ventilation only the mode AVAPS will be used if the patient is randomized in the experimental group.
| Study group | Role | What they receive |
|---|---|---|
| AVAPS | Experimental | Arm assigned to AVAPS mode for nocturnal ventilation with the same setting than bilevel pressure support but with AVAPS mode activated Receives: Device: Nocturnal ventilation |
| Bilevel pressure | Active comparator | Arm treated only with bilevel pressure support for nocturnal ventilation without activation of AVAPS mode Receives: Device: Nocturnal ventilation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 7 days
Secondary outcomes (6)
daytime arterial PCO2 after launching ventilation.
To evaluate efficacy of AVAPS Mode at day 90 on daytime arterial PCO2 after launching ventilation.
Measured: 90 days
Compliance to ventilation
To evaluate compliance (h/24h) to ventilation at days 7 and 90.
Measured: 7 and 90 days
Symptoms
To evaluate clinical efficacy on respiratory symptoms, dyspnea and sleepiness at day 1 and 90.
Measured: 90 days
Sleep studies
To evaluate effect of AVAPS on polysomnography, nocturnal SaO2, nocturnal PtCO2 at day 90.
Measured: 90 days
OBJECTIVE SLEEPINESS
To evaluate Multiple sleep latency and Maintenance of wakefulness tests at day 90.
Measured: 90 days
Respiratory parameters
To evaluate effect of AVAPS on respiratory parameters VC and mouth maximal pressures.
Measured: 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2012 (estimated)
- Study startJul 2012
- Primary completionMay 2016
- Study completionMay 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805
- Sponsor type
- University, hospital or other
- Collaborators
- Adep Assistance, Philips Respironics
- Responsible party
- Principal investigator, david orlikowski, Centre d'Investigation Clinique et Technologique 805
- Investigators
- David ORLIKOWSKI, Md-PhD, Principal investigator, Raymond Poincare Hospital - Garches - France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01530841
- Sponsor's protocol ID
- 2010-A01306-33
- Status last verified
- Mar 2017
- First submitted
- 8 Feb 2012
- Last updated
- 10 Mar 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01530841
- Acronym / sponsor ID
- 2010-A01306-33
- Conditions
- Myopathy
- Keywords
- Myotonic dystrophy type 1, Home ventilation, Respiratory insufficiency, Neuromuscular
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805University, hospital or other
- Collaborators
- Adep Assistance, Philips Respironics
- Study start
- Jul 2012
- Primary completion
- May 2016 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 10 Feb 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AVAPS | Experimental | Device: Nocturnal ventilationArm assigned to AVAPS mode for nocturnal ventilation with the same setting than bilevel pressure support but with AVAPS mode activated |
| Bilevel pressure | Active comparator | Device: Nocturnal ventilationArm treated only with bilevel pressure support for nocturnal ventilation without activation of AVAPS mode |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nocturnal ventilationAlso: BIPAP A30Home ventilation only the mode AVAPS will be used if the patient is randomized in the experimental group. | Device | AVAPS, Bilevel pressure |
Primary outcome
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
6 secondary outcomes
daytime arterial PCO2 after launching ventilation.
Time frame: 90 days
Compliance to ventilation
Time frame: 7 and 90 days
Symptoms
Time frame: 90 days
Sleep studies
Time frame: 90 days
OBJECTIVE SLEEPINESS
Time frame: 90 days
Respiratory parameters
Time frame: 90 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David ORLIKOWSKI, Md-PhD principal investigator
Raymond Poincare Hospital - Garches - France
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Nocturnal ventilation in people with Myopathy, run by Centre d'Investigation Clinique et Technologique 805 at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
arterial PCO2 under ventilation (time frame: 7 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2017.