About this trial
Hereditary neuropathies are a phenotypically and genetically heterogeneous group of disorders. One of the most common forms is Charcot-Marie-Tooth neuropathy (CMT), which can be further divided into demyelinating (CMT1) and axonal (CMT2) neuropathies, as well as various pathogenic genetic variants. In addition to the clinically predominant motor and sensory deficits, symptoms of the autonomic nervous system have also been described in patients with CMT, often leading to significant limitations in daily functioning and quality of life. However, little is known about the prevalence and extent of autonomic dysfunction in CMT patients.
In this study, patients with CMT will be assessed for the presence, severity, and characteristics of autonomic dysfunction using questionnaires and non-invasive diagnostic methods. Furthermore, diagnosis, genotype, and individual disease data-such as disease duration, severity of neurological impairment, and comorbidities-will be collected from patient records.
The aim of this study is to evaluate and characterize autonomic dysfunction in patients with CMT. It seeks to determine how frequently autonomic dysfunction occurs in CMT, which areas of the autonomic nervous system are most commonly affected, whether risk factors exist, and what differences can be observed between the various CMT subtypes. The findings of this study are expected to provide new insights into the role of autonomic dysfunction in CMT, ultimately contributing to improved care and treatment for affected patients.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
Clinical CMT Diagnosis / Anamnestically Healthy Control Group
Genetic confirmation of CMT in adult patients
Ability to achieve the outcome measure at baseline
Age between 18 and 65 years
Disqualifiers
Pregnancy or breastfeeding period
Other relevant neurological or psychiatric disorders, acute or in the past history
Presence of a serious previous internal disease
Trial population
Participants from the Biomarkers and Validation of Selected Outcome Measures (CMTNSmod) study (ClinicalTrials.gov ID NCT03386266)
Trial design
Other
Prospective
Treatments tested in this trial
Not listed
Trial groups
Trial outcomes
Primary outcomes
COMPASS 31
validated questionaire
electrophysiological measurement
Validated electrophysiological examinations including the sympathetic skin reflex, the Schellong test, and heart rate variability measurement
Secondary outcomes
muscle strength
neurography
Examinations measuring the electrical activity and conductivity of nerves, which are used to diagnose nerve damage or diseases.
nerve sonography
Sponsors and contacts
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