RecruitingNot Applicable

Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA

United States45 participantsStarted 2025-04-24

Plain-language 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.

Who can participate

Age range12 Years

SexALL

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Inclusion criteria

✓. 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 criteria

✕. 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)

What they're measuring

SMA EFFORT

Timeframe: Baseline, 1 week, 1 month

Trial details

NCT IDNCT06955897

SponsorColumbia University

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2026-12-07

Contact for this trial

Rafael Rodriguez-Torres, DPT

3472877093 rsr2157@cumc.columbia.edu

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