Status:
RECRUITING
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Lead Sponsor:
Columbia University
Collaborating Sponsors:
Biogen
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
12+ years
Brief Summary
The purpose of this project is to investigate the utility of the SMA EFFORT, an SMA-specific patient-reported outcome measure, to assess perceived physical fatigability that is anchored to intensity a...
Detailed Description
Fatigue and fatigability are symptoms often experienced by people living with spinal muscular atrophy (pwSMA). Nusinersen is shown to extend survival and improve motor function, but fatigue and fatiga...
Eligibility Criteria
Inclusion
- All types of 5qSMA (homozygous deletion or point mutation of the SMN1 gene and any number of SMN2 copies)
- Receiving a stable dosing regimen of 12mg nusinersen for at least 6 months
Exclusion
- An injury or surgery within the previous 3 months that would impact their ability to perform in-clinic function and/or fatigability assessments
- Enrolled in an ongoing clinical trial, or extension study, expanded access program, or long-term registry of an investigational or recently approved medication
- Receiving adjuvant and/or dual therapy (e.g., muscle-targeted, NMJ, or symptomatic treatments)
Key Trial Info
Start Date :
April 24 2025
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
February 26 2027
Estimated Enrollment :
45 Patients enrolled
Trial Details
Trial ID
NCT06955897
Start Date
April 24 2025
End Date
February 26 2027
Last Update
May 2 2025
Active Locations (1)
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1
Columbia University Irving Medical Center
New York, New York, United States, 10032