Muscular DystrophiesMyopathiesSpinal Muscular Atrophy (SMA)Charcot Marie Tooth Disease
Investigational drug(s) / intervention(s)
inapplicable
inapplicable: inapplicable
Study summary
This study has the general objective of observing walking parameters during a clinical test to objectively estimate fatigue in patients with neuromuscular diseases. Furthermore, the investigators want to evaluate the feasibility of collecting physical activity in daily life conditions during a one-week monitoring period using a wearable sensor.
Eligibility
Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
Accepted
Inclusion Criteria:
* Ambulant adult patients with genetic diagnosis of muscular dystrophy/myopathy (dystrophinopathies, muscular dystrophies and congenital and non-congenital myopathies), of spinal muscular atrophy (SMA) and with molecular diagnosis of Charcot-Marie Tooth 1 or 2.
* independent walking, even with assistance;
Exclusion Criteria:
* Dilated or ischemic heart disease with moderate impairment;
* Chronic respiratory failure: forced vital capacity (FVC) \< 40%; more than 5% of nocturnal time spent with peripheral oxygen saturation levels \< 90.
Primary outcome measure(s)
Variation in walking speed during a 6-minutes walking test — Baseline Variation in walking speed calculated as difference between the first and the final minute of the a 6-minutes walking test
Subjective fatigue — Baseline Evaluation of subjective fatigue using the Fatigue Severity Scale (FSS). The minimum value is 0, the maximum is 63. Higher scores mean worse outcome.
Trial sites (2)
Facility
City
Region
Status
Scientific Institute IRCCS E. Medea - Sede di Bosisio Parini
Bosisio Parini
Lecco
Recruiting
Scientific Institute IRCCS E. Medea - Polo di Conegliano
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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