Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Recruiting
Observational Study
Spinal Muscular Atrophy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 12 and older
- Sex
- Any
- Study type
- Observational
- Participants needed
- 45 (estimated)
- Sponsor
- Columbia University · Other
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About This Trial
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…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. All types of 5qSMA (homozygous deletion or point mutation of the SMN1 gene and any number of SMN2 copies)
2. Receiving a stable dosing regimen of 12 or 28 mg nusinersen for at least 6 months
Exclusion Criteria:
1. An injury or surgery within the previous 3 months that would…
Contacts