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Screening Questionnaire for Respiratory Muscle Weakness and Sleep-disordered Breathing in Neuromuscular Disorders

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ClinicalTrials.gov Identifier: NCT02833168
Recruitment Status : Recruiting
First Posted : July 14, 2016
Last Update Posted : July 14, 2016
Sponsor:
Information provided by (Responsible Party):
Matthias Boentert, Westfälische Wilhelms-Universität Münster

Brief Summary:
It is the aim of this project to develop and validate a German language screening questionnaire for symptoms of respiratory muscle weakness and sleep-disordered breathing (SDB) in patients with neuromuscular disorders.

Condition or disease Intervention/treatment
Neuromuscular Disorders Other: patient-filled questionnaires Device: spiromanometry

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 : Observational
Estimated Enrollment : 90 participants
Observational Model: Case Control
Time Perspective: Prospective
Official Title: Validation of a German Language Screening Questionnaire for Symptoms of Respiratory Muscle Weakness and Sleep-disordered Breathing in Patients With Neuromuscular Disorders
Study Start Date : April 2016
Estimated Primary Completion Date : December 2016
Estimated Study Completion Date : December 2016

Resource links provided by the National Library of Medicine


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.
Other: patient-filled questionnaires
All patients answer selected questionnaires on sleep-related symptoms, sleep quality, daytime performance, and health-related quality of life.

Device: spiromanometry
Measurement of forced vital capacity, maximum inspiratory pressure and maximum expiratory pressure

2
Patient with proven obstructive sleep apnea syndrome prior to CPAP initiation.
Other: patient-filled questionnaires
All patients answer selected questionnaires on sleep-related symptoms, sleep quality, daytime performance, and health-related quality of life.

Device: spiromanometry
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.
Other: patient-filled questionnaires
All patients answer selected questionnaires on sleep-related symptoms, sleep quality, daytime performance, and health-related quality of life.

Device: spiromanometry
Measurement of forced vital capacity, maximum inspiratory pressure and maximum expiratory pressure




Primary Outcome Measures :
  1. Nocturnal hypercapnia tcCO2 [ Time Frame: 1 day ]


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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with neuromuscular disorders, newly diagnosed obstructive sleep apnea syndrome or sleep disorders other than sleep-related breathing disorders (30 patients in each group)
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02833168


Contacts
Contact: Matthias Boentert, MD +49-251-83 ext 44458 matthias.boentert@ukmuenster.de
Contact: Peter Young, MD +49-251-83 ext 48196 peter.young@ukmuenster.de

Locations
Germany
University Hospital Münster, Department of Sleep Medicine and Neuromuscular Disorders Recruiting
Münster, Germany, 48149
Contact: Matthias Boentert, MD    +49-251-83 ext 44458    matthias.boentert@ukmuenster.de   
Contact: Peter Young, MD    +49-251-83 ext 48196    peter.young@ukmuenster.de   
Principal Investigator: Matthias Boentert, MD         
Sub-Investigator: Anna Heidbreder, MD         
Bethanien Hospital Recruiting
Solingen, Germany, 42699
Contact: Winfried Randerath, MD    +49-212 630 ext 0    Winfried.Randerath@klinik-bethanien.de   
Contact: Wiebke Dohrn, MD    +49-212 630 ext 0    Wiebke.Dohrn@klinik-bethanien.de   
Sponsors and Collaborators
Westfälische Wilhelms-Universität Münster
Investigators
Study Chair: Matthias Boentert, MD University Hospital Münster, Department of Sleep Medicine and Neuromuscular Disorders

Responsible Party: Matthias Boentert, MD, Senior Consultant in Neurology and Sleep Medicine, Westfälische Wilhelms-Universität Münster
ClinicalTrials.gov Identifier: NCT02833168     History of Changes
Other Study ID Numbers: KSN_2016_1_MB
First Posted: July 14, 2016    Key Record Dates
Last Update Posted: July 14, 2016
Last Verified: July 2016

Keywords provided by Matthias Boentert, Westfälische Wilhelms-Universität Münster:
Neuromuscular disorders
Sleep disordered breathing
Dyspnea

Additional relevant MeSH terms:
Disease
Respiratory Aspiration
Muscle Weakness
Paresis
Neuromuscular Diseases
Pathologic Processes
Respiration Disorders
Respiratory Tract Diseases
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms