MyopathyPhase 3DYVINENCT01225614
Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive
A Phase 3 study testing Bilevel pressure ventilator in people with Myopathy and Muscular Weakness, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
This is a multicenter randomized controlled open labeled study testing efficacy and tolerance of early launching of night non invasive ventilation in patients with myotonic dystrophy type 1(DM1). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myopathy, Muscular Weakness, Respiratory Insufficiency
- What is being tested?
- Bilevel pressure ventilator
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 77 participants
- Where
- 1 siteFrance
- Study dates
- Oct 2010to Dec 2018
- Phase
- Phase 3
- 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
- Man or woman of age ≥ 18 years
- Preliminary medical examination
- Enlightened and written consent
- Genetically proved Steinert disease
- Presenting at least one of the following 3 criteria
- A hypercapnia: PaCO2 > 45 mmHg or
Show all 9Show fewer
- A night-desaturation: SaO2 < 88 % more than consecutive 5 minutes or
- Apnea syndrome with significant sleep:index of apnea / hypopnea> a 30 / hour
- And with presence of at least a clinical sign: dyspnoea, orthopnea, headaches, asthenia, diurnal sleepiness, or any other sign suggestive of disturbance of the sleep or of respiratory dysfunction
Who cannot join
- Age inferior to 18
- Regime of legal protection
- Pregnancy
- Absolute indication for ventilation: clinical signs (dyspnoea, orthopnea, headaches, asthenia, diurnal sleepiness), AND PaCO2 > 60 mmHg, AND night-desaturation < 88 % AND one CV < 50 % of the theoretical or the PIMAX < 60 cm H2O
- Acute respiratory failure
- Already ventilated patient
- Patient under oxygen
- Not (beneficiary to a regime of Social Security or legal successor)
Trial locations and contacts
1 site in 1 country.
France1 site
Home ventilation unit and intensive care, centre of neuromuscular disease (Garches Mondor Necker Hendaye), Raymond Poincaré hospital Versailles Saint Quentin University.
Garches, 92380
About this study
Official title: Study of Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive Ventilation in Adults With Myotonic Dystrophy Type 1(DM1)
This is a multicenter randomized controlled open labeled study testing efficacy and tolerance of early launching of night non invasive ventilation in patients with myotonic dystrophy type 1(DM1). The object of this project is to estimate the effects of the early introduction of non invasive ventilation on the arisen of complication (non expected hospitalization, tracheostomy even death) with regard to a simple respiratory follow-up in patients affected by myotonic dystrophy.
Read the detailed description
DM1 is the most frequent genetic myopathy in the adult. Actually there is no curative treatment, and symptomatic cares are essentials. The respiratory impairment is the main cause of morbid-mortality at these patients. The median of survival of the patients affected by DM1 with respiratory failure is of 59 years. Mechanisms of disease are complex implying a central and a direct impairment of respiratory muscles. These patients can present an alveolar hypoventilation, notably during night, not correlated to the muscular weakness. These patients often present a cognitive impairment complicating the interpretation of the clinical symptoms and their compliance to treatments. International recommendations for launching mechanical ventilation in neuromuscular diseases are the presence: 1) at least a clinical sign of alveolar hypoventilation, and one of the following criteria 2) diurnal hypercapnia (> 45 mmHg), 3) restrictive syndrome (vital capacity < 50 % and\or maximal inspiratory pressure < 60 cmH20), 4) the existence of a oxygen night-desaturation (SaO2 < 88 %) of more than 5 minutes. However, a Cochrane meta analyzes underline the absence of randomised study estimating the profit risk in the long term of the night-mechanical ventilation for progressive myopathies such as the DM1. The validity of these criteria and the effect of the ventilation on the survival and the complications were never estimated in DM1. On a retrospective series, the compliance is inferior and the observance is only 20 % a year and the incidence of the complications (death or resort to a tracheostomy) was 6 times as important in non observant patients.
Objective (s) of the clinical study To estimate the efficiency and the tolerance of long term night-non invasive mechanical ventilation in patients affected by DM1.
Main judgment criteria:
Mortality and non programmed hospitalization.
Experimental plan:
Multicenter, national, randomized, controlled, study on 2 parallel groups. The subjects presenting a theoretical indication following consensual criteria of ventilation will be randomized either for a start up of ventilation or for an annual monitoring.
Hypothesis: Early starting of non invasive ventilation allows a reduction of 20 % of the mortality or the number of non-programmed hospitalization compared to the control group for which the rate would be 40 %.
- Conditions
- Myopathy, Muscular Weakness, Respiratory Insufficiency
- Being tested
- Bilevel pressure ventilator
- Keywords
- Home ventilation, Myotonic dystrophy type 1 (DM1)
- Medical subject headings
- Muscular Diseases, Muscle Weakness, Respiratory Insufficiency, Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bilevel pressure ventilatorDevice
Nocturnal home ventilation
| Study group | Role | What they receive |
|---|---|---|
| bipap ventilation | Experimental | Not described Receives: Device: Bilevel pressure ventilator |
| Standard care | Active comparator | Standard care and ventilation if occurrence of absolute criteria of ventilation (cf infra). Receives: Device: Bilevel pressure ventilator |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.
Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.
Measured: 5 YEARS
Secondary outcomes (7)
Distribution of survival between the randomisation at 5 years
Distribution of survival between the randomisation at 5 years
Measured: 5 YEARS
Number of patients having a formal indication of ventilation
Number of patients having a formal indication of ventilation
Measured: 5 YEARS
Number of tracheostomized patients at 5 years
Number of tracheostomized patients at 5 years
Measured: 5 YEARS
Number of non expected hospitalizations at 5 years
Number of non expected hospitalizations at 5 years
Measured: 5 YEARS
Observance of the ventilation defined by an average minimal use of 4 am by 12:00 pm .
Observance of the ventilation defined by an average minimal use of 4 am by 12:00 pm (determined in a objective way by the counter of the device).
Measured: 5 YEARS
Degree of respiratory and sleep impairment at 5 years
Degree of respiratory and sleep impairment at 5 years
Measured: 5 YEARS
Quality of life SF36, scales of depression
Quality of life SF36, scales of depression
Measured: 5 YEARS
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2010 (estimated)
- Study startOct 2010
- Primary completionJul 2018 (estimated)
- Study completionDec 2018 (estimated)
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.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 77 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Association Française contre les Myopathies (AFM), Paris
- Responsible party
- Sponsor
- Investigators
- DAVID ORLIKOWSKI, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01225614
- Sponsor's protocol ID
- P081221
- Other IDs
- 2009-A01023-54 (IDRCB)
- Acronym
- DYVINE
- Status last verified
- Nov 2017
- First submitted
- 20 Oct 2010
- Last updated
- 20 Nov 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive Ventilation in Adults With Myotonic Dystrophy Type 1(DM1)
- ClinicalTrials.gov ID
- NCT01225614
- Acronym / sponsor ID
- DYVINE / P081221
- Conditions
- Myopathy, Muscular Weakness, Respiratory Insufficiency
- Keywords
- Home ventilation, Myotonic dystrophy type 1 (DM1)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 77 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Association Française contre les Myopathies (AFM), Paris
- Study start
- Oct 2010 (actual)
- Primary completion
- Jul 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 21 Oct 2010 (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.
This is a multicenter randomized controlled open labeled study testing efficacy and tolerance of early launching of night non invasive ventilation in patients with myotonic dystrophy type 1(DM1). The object of this project is to estimate the effects of the early introduction of non invasive ventilation on the arisen of complication (non expected hospitalization, tracheostomy even death) with regard to a simple respiratory follow-up in patients affected by myotonic dystrophy.
Detailed description
DM1 is the most frequent genetic myopathy in the adult. Actually there is no curative treatment, and symptomatic cares are essentials. The respiratory impairment is the main cause of morbid-mortality at these patients. The median of survival of the patients affected by DM1 with respiratory failure is of 59 years. Mechanisms of disease are complex implying a central and a direct impairment of respiratory muscles. These patients can present an alveolar hypoventilation, notably during night, not correlated to the muscular weakness. These patients often present a cognitive impairment complicating the interpretation of the clinical symptoms and their compliance to treatments. International recommendations for launching mechanical ventilation in neuromuscular diseases are the presence: 1) at least a clinical sign of alveolar hypoventilation, and one of the following criteria 2) diurnal hypercapnia (> 45 mmHg), 3) restrictive syndrome (vital capacity < 50 % and\or maximal inspiratory pressure < 60 cmH20), 4) the existence of a oxygen night-desaturation (SaO2 < 88 %) of more than 5 minutes. However, a Cochrane meta analyzes underline the absence of randomised study estimating the profit risk in the long term of the night-mechanical ventilation for progressive myopathies such as the DM1. The validity of these criteria and the effect of the ventilation on the survival and the complications were never estimated in DM1. On a retrospective series, the compliance is inferior and the observance is only 20 % a year and the incidence of the complications (death or resort to a tracheostomy) was 6 times as important in non observant patients.
Objective (s) of the clinical study To estimate the efficiency and the tolerance of long term night-non invasive mechanical ventilation in patients affected by DM1.
Main judgment criteria:
Mortality and non programmed hospitalization.
Experimental plan:
Multicenter, national, randomized, controlled, study on 2 parallel groups. The subjects presenting a theoretical indication following consensual criteria of ventilation will be randomized either for a start up of ventilation or for an annual monitoring.
Hypothesis: Early starting of non invasive ventilation allows a reduction of 20 % of the mortality or the number of non-programmed hospitalization compared to the control group for which the rate would be 40 %.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| bipap ventilation | Experimental | Device: Bilevel pressure ventilator |
| Standard care | Active comparator | Device: Bilevel pressure ventilatorStandard care and ventilation if occurrence of absolute criteria of ventilation (cf infra). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Man or woman of age ≥ 18 years
- Preliminary medical examination
- Enlightened and written consent
- Genetically proved Steinert disease
Key exclusion criteria (8 listed)
- Age inferior to 18
- Regime of legal protection
- Pregnancy
- Absolute indication for ventilation: clinical signs (dyspnoea, orthopnea, headaches, asthenia, diurnal sleepiness), AND PaCO2 > 60 mmHg, AND night-desaturation < 88 % AND one CV < 50 % of the theoretical or the PIMAX < 60 cm H2O
- Acute respiratory failure
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 |
|---|---|---|
| Bilevel pressure ventilatorNocturnal home ventilation | Device | Standard care, bipap ventilation |
Primary outcome
Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.
Time frame: 5 YEARS
Rate of patients having a complication (number of non expected hospitalization or death) at 5 years.
7 secondary outcomes
Distribution of survival between the randomisation at 5 years
Time frame: 5 YEARS
Number of patients having a formal indication of ventilation
Time frame: 5 YEARS
Number of tracheostomized patients at 5 years
Time frame: 5 YEARS
Number of non expected hospitalizations at 5 years
Time frame: 5 YEARS
Observance of the ventilation defined by an average minimal use of 4 am by 12:00 pm .
Time frame: 5 YEARS
Degree of respiratory and sleep impairment at 5 years
Time frame: 5 YEARS
Quality of life SF36, scales of depression
Time frame: 5 YEARS
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 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
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Bilevel pressure ventilator in people with Myopathy and Muscular Weakness, run by Assistance Publique - Hôpitaux de Paris 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 9 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 20 Nov 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Rate of patients having a complication (number of non expected hospitalization or death) at 5 years. (time frame: 5 YEARS). 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 20 Nov 2017.