Trial document




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  DRKS00013905

Trial Description

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Title

Effects of Nasal-High Flow therapy on nocturnal hypoventialtion and autonomic nervous system function in patients with neuromuscular diseases

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Trial Acronym

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URL of the Trial

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Brief Summary in Lay Language

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Brief Summary in Scientific Language

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).
Mildly affected patients with an indication for NIV are often characterised by adherence problems although they would profit from it in terms of an early support for their diaphragmatic weakness and shortness of breath as a result of this.
Nasal High Flow (NHF) therapy presents a promising therapy that supplies warm heated humidified air with a high flow rate and hence also produces an intrinsic positive airway pressure.
The use of NHF has not been systematically investigated in patients with NME although NHF has been shown to reduce work of breathing in patients with primary lung diseases.
This study therefore aims to investigate the night time effects of NHF on respiration (hypoventilation), autonomic nervous system function and hemodynamics in patients with NIV treated hypoventilation due to NME. The effects of different Flow rates will be compared and effects will also be compared to effects of NIV itself.
In accordance with a sample size calculation conducted by the Institute of Medical Biometrics of Münster University Hospital (expert opinion obtained on December, 19th 2017) this will be done in a total of 15 patients with well-characterized NME.

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Organizational Data

  •   DRKS00013905
  •   2018/04/05
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  •   yes
  •   Approved
  •   2017-668-f-S, Ethik-Kommission der Ärztekammer Westfalen-Lippe und der med. Fakultät der Westfälischen Wilhelms-Universität Münster
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Secondary IDs

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Health Condition or Problem studied

  •   G12 -  Spinal muscular atrophy and related syndromes
  •   E74 -  Other disorders of carbohydrate metabolism
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Interventions/Observational Groups

  •   At night time 15 patients with an ongoing nocturnal NIV (Non-invasive ventilation) therapy for hypoventilation due to a NMD (Numerous neuromuscular disorders) will be attached to a full cardiorespiratory polysomnography in our university sleep lab. Beyond that they will be attached to a non-invasive hemodynamic monitor that continuously measures heart rate, blood pressure (including their variabilities) and bio-impedance based stroke volume.
    In the following night patients will be exposed to a split night investigations (in a randomized order) exposing them to three different interventions NIV, Nasal High Flow at a flow rate of 20 Liters/min and Nasal High Flow at a Flow rate of 50 Liters /min.
    The above mentioned interventions will be randomized in their order.
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Characteristics

  •   Interventional
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  •   Single arm study
  •   Open (masking not used)
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  •   Uncontrolled/Single arm
  •   Treatment
  •   Single (group)
  •   N/A
  •   N/A
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Primary Outcome

Transcutaneous CO2 [mmHG]
-> measured at night and under the above mentioned interventions

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Secondary Outcome

Heart rate [bpm], systolic blood pressure [mmHG], diastolic blood pressure [mmHG], stroke volume index [L/min/m²], high frequency component of diastolic blood pressure variability[%], low frequency component of heart rate variability [%], respiratory rate [/min], tidal volume [ml], minute ventilation [L/min], oxygen saturation [%], high frequency component of heart rate variability [%], low frequency component of diastolic blood pressure variability [%]
-> measured at night and under the above mentioned interventions

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Countries of Recruitment

  •   Germany
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Locations of Recruitment

  • University Medical Center 
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Recruitment

  •   Actual
  •   2018/05/01
  •   30
  •   Monocenter trial
  •   National
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Inclusion Criteria

  •   Both, male and female
  •   18   Years
  •   no maximum age
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Additional Inclusion Criteria

a. Age > 18 years (no age limit)
b. able to consent
c. proven NMD
d. ongoing nocturnal NIV therapy with a nasal mask for hypoventilation due to a NMD
e. good tolerance of and adherence to the ongoing NIV therapy with minimal nasal mask-related problems/discomfort
f. no vital indication for NIV use at night

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Exclusion Criteria

- Body Mass Index (BMI) > 35
- Heart failure with left ventricular ejection fraction <50%
- Chronic obstructive pulmonary disease, emphysema
- Severe psychiatric disease including dementia
- Thoracic wall disease
- Cardiac pacemaker

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Addresses

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    • Department für Neurologie - Klinik für Schlafmedizin und neuromuskuläre Erkrankungen Universitätsklinikum Münster
    • Albert-Schweitzer-Campus 1
    • 48149  Münster
    • Germany
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    • Leiter der Sektion PneumologieMedizinische Klinik IUniversitätsklinikum Aachen
    • Mr.  Univ.-Prof. Dr.  Michael  Dreher 
    • Pauwelsstraße 30
    • 52074  Aachen
    • Germany
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    • Department für Neurologie - Klinik für Schlafmedizin und neuromuskuläre Erkrankungen Universitätsklinikum Münster
    • Mr.  Dr. med.  Jens  Spiesshoefer 
    • Albert-Schweitzer-Campus 1
    • 48149  Münster
    • Germany
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    • Department für Neurologie- Klinik für Schlafmedizin und neuromuskuläre Erkrankungen Universitätsklinikum Münster
    • Mr.  Dr. med.  Matthias  Boentert 
    • Albert-Schweitzer-Campus 1
    • 48149  Münster
    • Germany
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Sources of Monetary or Material Support

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    • Department für Neurologie - Klinik für Schlafmedizin und neuromuskuläre Erkrankungen Universitätsklinikum Münster
    • Albert-Schweitzer-Campus 1
    • 48149  Münster
    • Germany
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Status

  •   Recruiting ongoing
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Trial Publications, Results and other Documents

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