Myotonic DystrophyNCT01242007
Postural Spirometry Changes in Ambulatory Myotonic Dystrophy Patients
A study testing Supine spirometry in people with Myotonic Dystrophy, run by University of Nancy at 1 site in France.
Trial summary
Myotonic dystrophy Type 1 (MD1, Steinert's disease), an autosomal dominant multisystem disease, is of the most common muscular dystrophies in adults, with a European prevalence of 3-15/100 000. The disease course is progressive, associating muscular weakness, wasting and myotonia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myotonic Dystrophy
- What is being tested?
- Supine spirometry
- How many people
- 58 participants
- Where
- 1 siteFrance
- Study dates
- Apr 2008to Jun 2010
- 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
- clinical diagnosis of myotonic dystrophy type 1
- 18 years of age and older
- must be able to perform reproducible ventilatory manoeuvres
Who cannot join
- required non-invasive ventilation
- non reproducible spirometry results
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Lung Function Impairment and Postural Spirometry Changes in Ambulatory Myotonic Dystrophy Patients
Myotonic dystrophy Type 1 (MD1, Steinert's disease), an autosomal dominant multisystem disease, is of the most common muscular dystrophies in adults, with a European prevalence of 3-15/100 000. The disease course is progressive, associating muscular weakness, wasting and myotonia. Respiratory dysfunction is common, involving a restrictive ventilatory abnormality and alveolar hypoventilation, originating from respiratory muscle weakness. Depending on the degree of impairment of their lung function, the quality of life and the prognosis of MD1 patients may be very variable. However, time course and prevalence of such respiratory function impairment have not been clearly identified. More importantly, factors able to predict poor respiratory outcome have not been defined and therefore early prognosis can not be assessed during the follow-up of these patients. In other neuromuscular disorders, especially Amyotrophic Lateral Sclerosis (ALS), postural spirometry has been recommended to improve the detection of diaphragmatic involvement and some authors have suggested that the supine fall in the forced vital capacity could be used to initiate noninvasive positive pressure ventilation and predicts some respiratory symptoms.
In a sample of ambulatory patients with MD1, our study was designed to prospectively achieve two aims: 1) to assess the respective prevalence of a ventilatory restrictive pattern, respiratory muscle weakness, hypoxemia and hypercapnia and 2) to evaluate whether postural changes in lung volumes contribute to sensitize the diagnosis of respiratory weakness and could be used as a predictor of poor respiratory function, including hypoxemia, hypercapnia and restrictive ventilatory disease.
Read the detailed description
Materials and Methods :
Subjects:
Adult ambulatory patients (18 years of age and older) with a clinical diagnosis of myotonic dystrophy type I were investigated prospectively as part of routine follow-up, from april 2008 to june 2010. Patients were clinically evaluated in the department of "Internal Medicine" and lung function was assessed in the department of "Pulmonary Function Testing", both from the University Hospital of Nancy. Pulmonary tests were ordered for clinical indications, not part of a study protocol. The supine evaluation was added of the conventional lung function testing. All individual were examined and categorized according to a standardized five-point muscular-impairment rating scale, in which a score of 1 indicates no muscular impairment, 2 minimal signs without distal weakness except for digit flexors, 3 distal weakness without proximal weakness except for elbow extensors, 4 moderate proximal weakness, and 5 severe weakness (MIRS).
Lung and respiratory muscle function:
All pulmonary function tests met or exceeds applicable standards of the European Respiratory Society / American Thoracic Society.
Spirometry was performed in the upright-seated position and in the supine position. Respiratory function data were compared with the predicted normal values obtained by the European Community for Steel and Coal and expressed as percentage of the normal value. The flow/volume curve and lung volumes were respectively assessed by an open-circuit spirometry and plethysmography.
Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) were both measured in the seated position using a standard flanged mouthpiece.MIP was measured from Residual Volume (RV) and MEP was measured from Total Lung Capacity (TLC), both in a standard manner. The manoeuvres were repeated at least three times, or until two identical readings were obtained, and the best value was taken. Respiratory Muscle Strength (RMS) was defined as the mean of MIP and MEP expressed as a percent of the predicted values.
Arterial sampling and blood gas analysis :
Arterial blood gases were drawn at rest from the radial artery of the nondominant arm while the patient was comfortably seated for at least 10 minutes. A sterile, self-filling and disposable pre-heparinized system was used to take 1.5 ml of arterial blood.
Arterial oxygen partial pressure (PaO2) and arterial carbon dioxide partial pressure (PaCO2) were determined within 10 minutes after sampling. Room temperature and barometric pressure were recorded on a daily basis and were used to adjust calibrations and measurements. Quality control of the blood-gas equipment was performed twice a day, using standard solution.
- Conditions
- Myotonic Dystrophy
- Being tested
- Supine spirometry
- Keywords
- Myotonic dystrophy, Steinert's disease
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Supine spirometryOther
Also called Supine lung function testing
In addition to the current upright lung function evaluation we performed a supine spirometry
What is being measured
The primary outcome is the main question the study is designed to answer.
Evidence of lung function impairment
Evidence of ventilatory restriction assessed by lung function testing or hypoxemia or hypercapnia assessed by arterial blood gases analysis
Measured: 1 year on average (annual regular follow-up)
Secondary outcomes (1)
predictive factors of lung function impairment
Using results of upright and supine spirometry we intend to define variables that could predict poor respiratory outcome
Measured: 1 year on average (annual regular follow-up)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2010 (estimated)
- Study startApr 2008
- Primary completionJun 2010
- Study completionJun 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic DystrophyStudies 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
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Nancy
- Sponsor type
- University, hospital or other
- Collaborators
- Central Hospital, Nancy, France
- Investigators
- Bruno Chenuel, MD, PhD, Principal investigator, University of Nancy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01242007
- Sponsor's protocol ID
- UNBC-11102010
- Status last verified
- Nov 2010
- First submitted
- 15 Nov 2010
- Last updated
- 16 Nov 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 Nov 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Lung Function Impairment and Postural Spirometry Changes in Ambulatory Myotonic Dystrophy Patients
- ClinicalTrials.gov ID
- NCT01242007
- Acronym / sponsor ID
- UNBC-11102010
- Conditions
- Myotonic Dystrophy
- Keywords
- Myotonic dystrophy, Steinert's disease
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 58 participants (actual)
- Lead sponsor
- University of NancyUniversity, hospital or other
- Collaborators
- Central Hospital, Nancy, France
- Study start
- Apr 2008
- Primary completion
- Jun 2010 (actual)
- Study completion
- Jun 2010 (actual)
- First posted
- 16 Nov 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.
Myotonic dystrophy Type 1 (MD1, Steinert's disease), an autosomal dominant multisystem disease, is of the most common muscular dystrophies in adults, with a European prevalence of 3-15/100 000. The disease course is progressive, associating muscular weakness, wasting and myotonia. Respiratory dysfunction is common, involving a restrictive ventilatory abnormality and alveolar hypoventilation, originating from respiratory muscle weakness. Depending on the degree of impairment of their lung function, the quality of life and the prognosis of MD1 patients may be very variable. However, time course and prevalence of such respiratory function impairment have not been clearly identified. More importantly, factors able to predict poor respiratory outcome have not been defined and therefore early prognosis can not be assessed during the follow-up of these patients. In other neuromuscular disorders, especially Amyotrophic Lateral Sclerosis (ALS), postural spirometry has been recommended to improve the detection of diaphragmatic involvement and some authors have suggested that the supine fall in the forced vital capacity could be used to initiate noninvasive positive pressure ventilation and predicts some respiratory symptoms.
In a sample of ambulatory patients with MD1, our study was designed to prospectively achieve two aims: 1) to assess the respective prevalence of a ventilatory restrictive pattern, respiratory muscle weakness, hypoxemia and hypercapnia and 2) to evaluate whether postural changes in lung volumes contribute to sensitize the diagnosis of respiratory weakness and could be used as a predictor of poor respiratory function, including hypoxemia, hypercapnia and restrictive ventilatory disease.
Detailed description
Materials and Methods :
Subjects:
Adult ambulatory patients (18 years of age and older) with a clinical diagnosis of myotonic dystrophy type I were investigated prospectively as part of routine follow-up, from april 2008 to june 2010. Patients were clinically evaluated in the department of "Internal Medicine" and lung function was assessed in the department of "Pulmonary Function Testing", both from the University Hospital of Nancy. Pulmonary tests were ordered for clinical indications, not part of a study protocol. The supine evaluation was added of the conventional lung function testing. All individual were examined and categorized according to a standardized five-point muscular-impairment rating scale, in which a score of 1 indicates no muscular impairment, 2 minimal signs without distal weakness except for digit flexors, 3 distal weakness without proximal weakness except for elbow extensors, 4 moderate proximal weakness, and 5 severe weakness (MIRS).
Lung and respiratory muscle function:
All pulmonary function tests met or exceeds applicable standards of the European Respiratory Society / American Thoracic Society.
Spirometry was performed in the upright-seated position and in the supine position. Respiratory function data were compared with the predicted normal values obtained by the European Community for Steel and Coal and expressed as percentage of the normal value. The flow/volume curve and lung volumes were respectively assessed by an open-circuit spirometry and plethysmography.
Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) were both measured in the seated position using a standard flanged mouthpiece.MIP was measured from Residual Volume (RV) and MEP was measured from Total Lung Capacity (TLC), both in a standard manner. The manoeuvres were repeated at least three times, or until two identical readings were obtained, and the best value was taken. Respiratory Muscle Strength (RMS) was defined as the mean of MIP and MEP expressed as a percent of the predicted values.
Arterial sampling and blood gas analysis :
Arterial blood gases were drawn at rest from the radial artery of the nondominant arm while the patient was comfortably seated for at least 10 minutes. A sterile, self-filling and disposable pre-heparinized system was used to take 1.5 ml of arterial blood.
Arterial oxygen partial pressure (PaO2) and arterial carbon dioxide partial pressure (PaCO2) were determined within 10 minutes after sampling. Room temperature and barometric pressure were recorded on a daily basis and were used to adjust calibrations and measurements. Quality control of the blood-gas equipment was performed twice a day, using standard solution.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- clinical diagnosis of myotonic dystrophy type 1
- 18 years of age and older
- must be able to perform reproducible ventilatory manoeuvres
Key exclusion criteria (2 listed)
- required non-invasive ventilation
- non reproducible spirometry results
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 |
|---|---|---|
| Supine spirometryAlso: Supine lung function testingIn addition to the current upright lung function evaluation we performed a supine spirometry | Other | – |
Primary outcome
Evidence of lung function impairment
Time frame: 1 year on average (annual regular follow-up)
Evidence of ventilatory restriction assessed by lung function testing or hypoxemia or hypercapnia assessed by arterial blood gases analysis
1 secondary outcome
predictive factors of lung function impairment
Time frame: 1 year on average (annual regular follow-up)
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 16 Nov 2010.
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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.
Bruno Chenuel, MD, PhD principal investigator
University of Nancy
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Supine spirometry in people with Myotonic Dystrophy, run by University of Nancy 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 3 inclusion and 2 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 16 Nov 2010. 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?
Evidence of lung function impairment (time frame: 1 year on average (annual regular follow-up)). 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 16 Nov 2010.