Measuring Tiredness From Activity in People With Spinal Muscular Atrophy
Studies a new questionnaire on fatigue in people with spinal muscular atrophy treated with nusinersen.
Plain-language title generated from the registry record
Registry title: Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Key facts
- Where
- New York, United StatesSee the site
Where in United States
- Columbia University Irving Medical CenterNew York, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Columbia University
- Trial code
- NCT06955897
- Record updated
- 14 October 2026
- Original description
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.Read full original descriptionShow less
Characterizing Perceived Physical Fatigability in Nusinersen-treated Adolescents and Adults: The SMA EFFORT
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.
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 12 or 28 mg 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).