- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02833168
Screening Questionnaire for Respiratory Muscle Weakness and Sleep-disordered Breathing in Neuromuscular Disorders
Validation of a German Language Screening Questionnaire for Symptoms of Respiratory Muscle Weakness and Sleep-disordered Breathing in Patients With Neuromuscular Disorders
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
SDB is a promiment clinical feature of various neuromuscular disorders including amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and myopathies such as myotonic dystrophy type 1, Pompe disease, and limb-girdle muscular dystrophies (LGMD). In ALS, SMA, LGMD and Pompe disease, SDB is usually caused by nocturnal alveolar hypoventilaton due to diaphragmatic weakness which manifests first during sleep and REM sleep in particular. SDB usually leads to sleep disruption, non-restorative sleep and daytime symptoms including morning headache, hypersomnolence, and increased neuromuscular fatigue. In patients with severe diaphragmatic involvement both symptoms of SDB and potential complications of respiratory muscle weakness substantially add to overall disease burden of the disease and decrease life span. Diagnosis of SDB is established by means of sleep studies which should ideally comprise polysomnography (PSG) and transcutaneous capnography. PSG with capnometry is time-consuming, expensive and not readily available for patients or referring physicians, respectively. For this reason it is desirable to thoroughly screen patients with neuromuscular disease for symptoms of SDB and respiratory muscle weakness. Results from a validated screening questionnaire could be used as an adjunctive to pulmonary function testing or spirometry results in order to identify patients in whom sleep studies should be performed. In addition, a screening questionnaire would facilitate early recognition of patients with SDB, enabling treating physicians to take appropriate steps to establish the diagnosis and to initiate non-invasive ventilation as early as possible.
Until now, there is no validated German language screening questionnaire for symptoms of respiratory muscle weakness and SDB. Steier et al. published an English language questionnaire which was validated as a screening tool in 33 patients with very different neuromuscular disorders which were predominantly neurogenic (Steier et al. 2011). In this study, SDB was defined by an apnea hypopnea index above 5 per hour. Nocturnal oxygen saturation and CO2 monitoring were not taken into account at all. In addition the questionnaire does not systematically cover sleep-related symptoms of SDB in detail (such as sleep disruption and morning headache) which have to be separated from daytime symptoms such as dyspnea or orthopnea, respectively.
Thus, the current project aims to correlate comprehensively generated items of a screening questionnaire with sleep study results including capnography alongside with respiratory muscle testing in patients with neuromuscular disorders. As control subjects, patients with newly diagnosed obstructive sleep apnea syndrome and sleep disorders other than sleep-related breathing disorders are enrolled in the study.
Study Type
Enrollment (Anticipated)
Contacts and Locations
Study Contact
- Name: Matthias Boentert, MD
- Phone Number: 44458 +49-251-83
- Email: matthias.boentert@ukmuenster.de
Study Contact Backup
- Name: Peter Young, MD
- Phone Number: 48196 +49-251-83
- Email: peter.young@ukmuenster.de
Study Locations
-
-
-
Münster, Germany, 48149
- Recruiting
- University Hospital Münster, Department of Sleep Medicine and Neuromuscular Disorders
-
Contact:
- Matthias Boentert, MD
- Phone Number: 44458 +49-251-83
- Email: matthias.boentert@ukmuenster.de
-
Contact:
- Peter Young, MD
- Phone Number: 48196 +49-251-83
- Email: peter.young@ukmuenster.de
-
Principal Investigator:
- Matthias Boentert, MD
-
Sub-Investigator:
- Anna Heidbreder, MD
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Solingen, Germany, 42699
- Recruiting
- Bethanien Hospital
-
Contact:
- Winfried Randerath, MD
- Phone Number: +49-212 630
- Email: Winfried.Randerath@klinik-bethanien.de
-
Contact:
- Wiebke Dohrn, MD
- Phone Number: +49-212 630
- Email: Wiebke.Dohrn@klinik-bethanien.de
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
Inclusion Criteria:
- proven neuromuscular disease known to be potentially associated with diaphragmatic weakness (group 1)
- newly diagnosed obstructive sleep apnea with an apnea hypopnea index > 15/h total sleep time (group 2)
- newly diagnosed narcolepsy, hypersomnia, insomnia or parasomnia in the absence of any sleep-related breathing disorder (group 3)
- availability of recent diagnostic sleep studies including polysomnography and transcutaneous capnography
Exclusion Criteria:
- ongoing CPAP oder non-invasive ventilation
- inability to participate in study procedures
- severe lung disease
Study Plan
How is the study designed?
Design Details
- Observational Models: Case-Control
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
1
Patients with proven neuromuscular disorders known to be potentially associated with significant diaphragmatic weakness, e. g.
ALS, myotonic dystrophy type 1, limb-girdle muscular dystrophy, Duchenne and Becker muscular dystrophy.
Patients already receiving home ventilatory support will not be included in the study.
|
All patients answer selected questionnaires on sleep-related symptoms, sleep quality, daytime performance, and health-related quality of life.
Measurement of forced vital capacity, maximum inspiratory pressure and maximum expiratory pressure
|
2
Patient with proven obstructive sleep apnea syndrome prior to CPAP initiation.
|
All patients answer selected questionnaires on sleep-related symptoms, sleep quality, daytime performance, and health-related quality of life.
Measurement of forced vital capacity, maximum inspiratory pressure and maximum expiratory pressure
|
3
Patients with sleep disorders other than sleep-related breathing disorders, e. g. narcolepsy, hypersomnia, parasomnia or sleep-related movement disorders.
|
All patients answer selected questionnaires on sleep-related symptoms, sleep quality, daytime performance, and health-related quality of life.
Measurement of forced vital capacity, maximum inspiratory pressure and maximum expiratory pressure
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Nocturnal hypercapnia tcCO2
Time Frame: 1 day
|
1 day
|
Collaborators and Investigators
Investigators
- Study Chair: Matthias Boentert, MD, University Hospital Münster, Department of Sleep Medicine and Neuromuscular Disorders
Study record dates
Study Major Dates
Study Start
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Respiratory Tract Diseases
- Apnea
- Respiration Disorders
- Sleep Disorders, Intrinsic
- Dyssomnias
- Sleep Wake Disorders
- Neurologic Manifestations
- Musculoskeletal Diseases
- Muscular Diseases
- Neuromuscular Manifestations
- Sleep Apnea Syndromes
- Disease
- Respiratory Aspiration
- Muscle Weakness
- Paresis
- Neuromuscular Diseases
Other Study ID Numbers
- KSN_2016_1_MB
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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