Clinical trial NCT06666816: Observational Study to Observe Variations of Gait Parameters in Patients with Neuromuscular Diseases
Official title: Observational Study for the Identification of Fatigue and Gait Biomarkers in Populations with Neuromuscular Pathologies in the Clinical Context and Daily Life
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Muscular Dystrophies
Myopathies
Spinal Muscular Atrophy (SMA)
Charcot Marie Tooth Disease
Lead sponsor: IRCCS Eugenio Medea
Sponsor class: OTHER
First posted: 2024-10-31
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Potentially eligible participants will be selected from clinical databases of IRCCS Medea

Brief 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 criteria: 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 outcomes:
measure: Variation in walking speed during a 6-minutes walking test
timeFrame: Baseline
measure: Subjective fatigue
timeFrame: Baseline
Secondary outcomes:
measure: Clinical progression of the neuromuscular disease
timeFrame: Baseline
measure: Clinical progression of the neuromuscular disease
timeFrame: Baseline
measure: Variation in gait variability during the 6-Minutes Walking Test
timeFrame: Baseline
Other outcomes:
measure: Acceptability of the activity monitoring device during daily life
timeFrame: Baseline
measure: User friendliness of the activity monitoring device during daily life
timeFrame: Baseline
measure: Physical activity in daily life
timeFrame: Baseline
Locations:
city: Bosisio Parini
country: Italy
facility: Scientific Institute IRCCS E. Medea - Sede di Bosisio Parini
state: Lecco
city: Conegliano
country: Italy
facility: Scientific Institute IRCCS E. Medea - Polo di Conegliano
state: Treviso
Study officials:
References:
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