Motor Function and Activity of Daily Life in Spinal Muscular Atrophy

Spinal Muscular Atrophy Clinical Trial

Official title:

Translation of Motor Function to Daily Activity Performance in Individuals With SMA

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05931211
• Other study ID #: 202305039RINB
• Status: Not yet recruiting
• Start date: June 30, 2023
• Est. completion date: December 31, 2024

Study information

• Verified date: May 2023
• Source: National Taiwan University Hospital
• Contact: Hsi-Wen Huang, MSc
• Phone: 886-2-23123456
• Email: lovelyeva39[@]gmail.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 100
• Est. completion date: December 31, 2024
• Est. primary completion date: June 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Locations

Country	Name	City	State
Taiwan	National Taiwan University Hospital	Taipei

Sponsors (1)

Lead Sponsor	Collaborator
National Taiwan University Hospital

Country where clinical trial is conducted

Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Revised Upper Limb Module	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.	15-20 minutes