Motor Function and Activity of Daily Life in Spinal Muscular Atrophy

Not yet recruiting

• Conditions: Spinal Muscular Atrophy

• Registration Number: NCT05931211
• Lead Sponsor: National Taiwan University Hospital

Brief Summary

This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.

Detailed Description

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease that causes progressive muscle weakness and atrophy and eventually leads to loss of function. Despite recent advances in disease-modifying treatments, SMA can still significantly impact an individual's functioning in daily living and quality of life. This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.

Study Timeline

• Status Verified: 2023-05
• Study First Submitted: 2023-06-26
• Study First Submitted QC: 2023-06-26
• Study Start: 2023-06-30
• Study First Posted: 2023-07-05
• Last Update Submitted: 2023-07-05
• Last Update Posted: 2023-07-07
• Primary Completion: 2024-06-30
• Study Completion: 2024-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

• Having a diagnosis of SMA with genetics confirmed as 5q SMA.
• Above 3 years of age (capable of performing motor assessments).

Exclusion Criteria

• Having severe respiratory and other medical conditions precluding safe participation in standardized motor function tests.
• Having any orthopedic surgery during the past 6 months.
• Women who are pregnant.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Revised Upper Limb Module	15-20 minutes	The physiotherapist will perform a serious of motor test to evaluate the patient's upper limb motor function. This test is to observe the manual ability on different tasks in sitting position, including drawing along the path, opening Ziploc container, or lifting weight. And the therapist will score for each item. The Revised Upper Limb Module (RULM) are consist of 19 items. It takes about 15-20 minutes for testing.

Trial Locations

Locations (1)

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan