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

Neuromuscular Disorders Clinical Trial

Official title:

Validation of a German Language Screening Questionnaire for Symptoms of Respiratory Muscle Weakness and Sleep-disordered Breathing in Patients With Neuromuscular Disorders

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02833168
• Other study ID #: KSN_2016_1_MB
• Status: Recruiting
• Phase: N/A
• First received: July 12, 2016
• Last updated: July 13, 2016
• Start date: April 2016
• Est. completion date: December 2016

Study information

• Verified date: July 2016
• Source: Westfälische Wilhelms-Universität Münster
• Contact: Matthias Boentert, MD
• Phone: +49-251-83
• Email: matthias.boentert[@]ukmuenster.de
• Is FDA regulated: No
• Health authority: Ethics Commission WWU Munster/Ärztekammer Westfalen/Lippe, Germany:
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 90
• Est. completion date: December 2016
• Est. primary completion date: December 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 80 Years

Eligibility

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 Design

Observational Model: Case Control, Time Perspective: Prospective

Related Conditions & MeSH terms

• Disease
• Muscle Weakness
• Neuromuscular Diseases
• Neuromuscular Disorders
• Paresis
• Respiratory Aspiration

Intervention

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

Locations

Country	Name	City	State
Germany	University Hospital Münster, Department of Sleep Medicine and Neuromuscular Disorders	Münster
Germany	Bethanien Hospital	Solingen

Sponsors (1)

Lead Sponsor	Collaborator
Westfälische Wilhelms-Universität Münster

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Nocturnal hypercapnia tcCO2		1 day	No