Motor Function and Activity of Daily Life in Spinal Muscular Atrophy
Translation of Motor Function to Daily Activity Performance in Individuals With SMA
1 other identifier
observational
100
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Jun 2023
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 26, 2023
CompletedStudy Start
First participant enrolled
June 30, 2023
CompletedFirst Posted
Study publicly available on registry
July 5, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
December 31, 2024
CompletedJuly 7, 2023
May 1, 2023
1 year
June 26, 2023
July 5, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
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
Eligibility Criteria
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease that causes progressive muscle weakness and atrophy and eventually leads to loss of function.
You may qualify if:
- Having a diagnosis of SMA with genetics confirmed as 5q SMA.
- Above 3 years of age (capable of performing motor assessments).
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
National Taiwan University Hospital
Taipei, 100, Taiwan
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Hsi-Wen Huang, MSc
National Taiwan University Hospital
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- CROSS SECTIONAL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 26, 2023
First Posted
July 5, 2023
Study Start
June 30, 2023
Primary Completion
June 30, 2024
Study Completion
December 31, 2024
Last Updated
July 7, 2023
Record last verified: 2023-05
Data Sharing
- IPD Sharing
- Will not share