Multisystem and Autonomic Complications of NMD on Long-term NIV

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

35 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-10-25

Study Completion Date

2021-05-20

Brief Summary

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This exploratory observational study evaluates autonomic function using measures including heart rate variability in adults with childhood-onset neuromuscular and neurological disorders (NMD) who use non-invasive ventilation (NIV) as part of their long-term care. Autonomic function will be correlated with measures of symptom burden, disease severity and complications.

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Detailed Description

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Childhood-onset neuromuscular and neurological disorders (NMD) are a diverse group of rare conditions in which muscle function is significantly impaired. Due to progress in NMD management over recent decades, particularly the use of non-invasive ventilation (NIV) to treat chronic respiratory failure, many individuals are surviving into adulthood and even middle age. However, older individuals are developing potentially fatal complications related to the heart e.g. sudden cardiac death, gastrointestinal (GI) tract and increasing dependence on NIV.

Previous studies largely in children and adolescents suggest individuals with NMD may develop problems of the autonomic nervous system (ANS), a network of nerves vital to controlling the body's short-term responses to internal and external stimuli. It is likely some complications of NMD are related to ANS abnormalities.

This will be an exploratory observational study using a cross-sectional design in which non-invasive tools will be used to evaluate autonomic function during wake and sleep. Individuals over 16 years of age with a confirmed NMD diagnosis will be recruited. Derived parameters of autonomic function including heart rate variability will be compared to values from an exploratory reference group of age- and sex-matched healthy adults. Autonomic function will also be correlated to measures of symptom burden, complications, quality of life and disease severity. Qualitative methods will be used to further evaluate the experience and perceptions of adults with NMD in making decisions about their personal and medical care, which become more complex with increasing age and disease severity.

Conditions

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Neuromuscular Diseases Neurologic Disorder

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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Patient group

60 participants ≥16 years of age with a confirmed diagnosis of a childhood-onset NMD

No intervention used

Intervention Type OTHER

No intervention used

Exploratory reference group

20 healthy adults ≥16 years of age

No intervention used

Intervention Type OTHER

No intervention used

Interventions

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No intervention used

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

Patient group:

* Age ≥16 years
* Confirmed diagnosis of childhood-onset NMD
* Able and willing to provide informed consent
* Requires non-invasive ventilation on an intermittent or continuous basis to manage respiratory failure

Reference group:

* Age ≥16 years
* No significant co-morbidities
* Not on any regular medications
* Able and willing to provide informed consent

Exclusion Criteria

* Inability to travel to hospital site
* Clinical instability or acute deterioration requiring hospitalisation or changes to medication in the previous 4 weeks
* A female of child-bearing potential who is pregnant or intends to become pregnant during study involvement

Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes