Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Characterizing Perceived Physical Fatigability in Nusinersen-treated Adolescents and Adults: The SMA EFFORT
Columbia University
45 participants
Apr 24, 2025
OBSERVATIONAL
Conditions
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 and duration of activities. We aim to characterize perceived physical fatigability (PPF) in a diverse cohort of people with SMA (pwSMA) and evaluate the change of PPF before and after nusinersen dosing.
Eligibility
Inclusion Criteria2
- 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 Criteria3
- 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)
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Interventions
Observational
Locations(1)
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NCT06955897