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Respiratory Muscle Strength and Function in Neuromuscular Disorders and Chronic Obstructive Pulmonary Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03032562
Recruitment Status : Unknown
Verified January 2017 by Matthias Boentert, Westfälische Wilhelms-Universität Münster.
Recruitment status was:  Recruiting
First Posted : January 26, 2017
Last Update Posted : January 26, 2017
Sponsor:
Information provided by (Responsible Party):
Matthias Boentert, Westfälische Wilhelms-Universität Münster

Tracking Information
First Submitted Date January 24, 2017
First Posted Date January 26, 2017
Last Update Posted Date January 26, 2017
Actual Study Start Date December 1, 2016
Estimated Primary Completion Date December 31, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 25, 2017)
  • Presence of daytime hypercapnia [ Time Frame: 1 day ]
    paCO2 > 45 mmHg
  • Presence of sleep-disordered breathing [ Time Frame: 1 day ]
    nocturnal tcCO2 > 50 mmHg, delta tcCO2 > 10 mmHg
Original Primary Outcome Measures Same as current
Change History No Changes Posted
Current Secondary Outcome Measures
 (submitted: January 25, 2017)
twitch transdiaphragmatic pressure [ Time Frame: 1 day ]
difference between intraesophageal and intragastral pressure after posterior magnetic stimulation of the phrenic nerves
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Respiratory Muscle Strength and Function in Neuromuscular Disorders and Chronic Obstructive Pulmonary Disease
Official Title Multimodal Measurement of Respiratory Muscle Strength and Function: Validation of Volitional and Non-volitional Tests With Regard to Nocturnal Hypoventilation in Patients With Neuromuscular Disorders and Chronic Obstructive Pulmonary Disease
Brief Summary

Using an extensive set of both volitional and non-volitional tests of respiratory muscle function and strength it is the aim of this study to

  • identify disease-specific patterns of respiratory muscle impairment in different NMD and COPD
  • establish which set of tests is predictive of sleep-disordered breathing or daytime hypercapnia in patients with NMD or COPD, respectively.
  • to investigate the decline of respiratory muscle function in patients with progressive NMD and COPD along with sleep studies and capnography
Detailed Description

Numerous neuromuscular disorders (NMD) are characterized not only by weakness of axial and limb muscles but also by respiratory muscle and upper airway involvement. Respiratory muscle weakness first manifests as sleep-disordered breathing (SDB). SDB in neuromuscular disease mainly comprises alveolar hypoventilation but upper airway collapse may also occur because pharyngeal muscle tone is impaired by both neuromuscular weakness and sleep.

Diaphragmatic weakness and myopathy are hallmarks of advanced obstructive pulmonary disease leading to even greater impairment of gas exchange in affected patients.

Non-invasive ventilation (NIV) should be initiated in patients with diaphragm weakness if significant SDB or hypercapnic respiratory failure is present. Indication criteria for NIV include measurement of spiromanometric parameters such as forced vital capacity (FVC), maximum inspiratory pressure (MIP), or sniff nasal inspiratory pressure (SNIP). These measurements are volitional, i. e. test results are dependent on individual patient effort and may show intraindividual variance which may not properly reflect disease progression or treatment effects, respectively. Associations between volitional and non-volitional methods of respiratory muscle strength testing have been reported mainly for healthy individuals but only scarcely in patients with specific NMD.

Non-volitional tests of respiratory muscle strength include phrenic nerve conduction studies, diaphragmatic ultrasound, and magnetic stimulation of the phrenic nerves in particular. Magnetic stimulation is usually combined with esophageal and gastric manometry yielding the twitch transdiaphragmatic pressure which closely reflects diaphragmatic strength.

It is desirable to obtain validated data on both volitional and non-volitional tests of respiratory muscle strength in various NMD. Validation has to be carried out with regard to both daytime hypercapnia and nocturnal retention of carbon dioxide because the latter often represents the earliest manifestation of respiratory muscle weakness. SDB often starts long before exertional dyspnea or orthopnea develop. Since NIV is one of the mainstays of symptomatic treatment in both NMD and advanced obstructive lung disease, early recognition of SDB is crucial for optimal patient care, and in many conditions, life expectancy and quality of life may be substantially increased by NIV. In this study, FVC, MIP, MEP and SNIP testing along with non-volitional methods and diaphragmatic ultrasound will be applied in order to establish a protocol which may help to reliably predict SDB or need for ventilatory support, respectively.

Patients with chronic obstructive pulmonary disease (COPD) develop flattening, shortening, and weakness of the diaphragm. This is not only due to downward dislocation of the diaphragm but also to impairment of contractile function, tissue structure, and intracellular biochemistry. Respiratory muscle weakness in COPD is of major clinical relevance since maximum inspiratory pressure is an independent predictor of survival in severely affected patients. In addition, hypercapnic respiratory failure due to inspiratory muscle weakness is associated with morbidity and mortality in COPD.

Direct comparison of both volitional and non-volitional measures of respiratory muscle function between patients with diaphragmatic weakness due to neuromuscular disease and patients with COPD is desirable. This approach may allow for further characterization of "impairment patterns" probably specific to either COPD or certain neuromuscular disease, respectively.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patients with known neuromuscular disease and individuals with chronic obstructive pulmonary disease (and no neuromuscular disorder)
Condition Respiratory Muscle Paralysis
Intervention Diagnostic Test: Multimodal measurement of respiratory muscle function and strength
Sleep-studies, capnography, spiromanometry, phrenic nerve conduction studies, magnetic phrenic nerve stimulation, diaphragmatic ultrasound
Study Groups/Cohorts
  • 1
    Patients with neuromuscular disease
    Intervention: Diagnostic Test: Multimodal measurement of respiratory muscle function and strength
  • 2
    Patients with chronic obstructive pulmonary disease
    Intervention: Diagnostic Test: Multimodal measurement of respiratory muscle function and strength
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Unknown status
Estimated Enrollment
 (submitted: January 25, 2017)
200
Original Estimated Enrollment Same as current
Estimated Study Completion Date February 28, 2020
Estimated Primary Completion Date December 31, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Patients with NMD (n=100): Amyotrophic lateral sclerosis, Pompe's disease, myotonic dystrophy type 1, limb-girdle muscular dystrophies, spinal muscular atrophy, etc.
  • patients with chronic obstructive lung disease (n=100)
  • age 18 - 80 years
  • written informed consent

Exclusion Criteria:

  • significant chest wall deformity
  • tracheostomy
  • inability to sit in the upright position
  • impaired consciousness
  • acute or severe medical comorbidity
  • mechanical ventilation for more than 4 hours during daytime
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers Not Provided
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Germany
Removed Location Countries  
 
Administrative Information
NCT Number NCT03032562
Other Study ID Numbers KSN_2016_2_MB
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Matthias Boentert, Westfälische Wilhelms-Universität Münster
Study Sponsor Westfälische Wilhelms-Universität Münster
Collaborators Not Provided
Investigators Not Provided
PRS Account Westfälische Wilhelms-Universität Münster
Verification Date January 2017