Observational Study to Observe Variations of Gait Parameters in Patients with Neuromuscular Diseases

Recruiting

• Conditions: Muscular Dystrophies; Myopathies; Spinal Muscular Atrophy (SMA); Charcot Marie Tooth Disease

• Registration Number: NCT06666816
• Lead Sponsor: IRCCS Eugenio 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-10-27
• Status Verified: 2024-10
• Study First Submitted: 2024-10-25
• Study First Submitted QC: 2024-10-29
• Last Update Submitted: 2024-10-29
• Study First Posted: 2024-10-31
• Last Update Posted: 2024-10-31
• Primary Completion: 2026-12
• Study Completion: 2026-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 120

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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.

Secondary Outcome Measures

Name	Time	Method
Clinical progression of the neuromuscular disease	Baseline	Progression of the neuromuscular disease using the Hammersmith Functional Motor Scale- Expanded (HFMS-E); the minimum value is 0, the maximum value is 69. Higher values mean better outcome.
Variation in gait variability during the 6-Minutes Walking Test	Baseline	Variation of step duration during the 6MWT extracted from a wearable inertial sensor. Measured in coefficient of variation (%). Higher values mean worse outcome.

Trial Locations

Locations (2)

Scientific Institute IRCCS E. Medea - Sede di Bosisio Parini; 🇮🇹 Bosisio Parini, Lecco, Italy

Scientific Institute IRCCS E. Medea - Polo di Conegliano; 🇮🇹 Conegliano, Treviso, Italy