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Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Characterizing Perceived Physical Fatigability in Nusinersen-treated Adolescents and Adults: The SMA EFFORT
Who can join
Ages 12 and older · All sexes
Full 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 impact their ability to perform in-clinic function and/or fatigability assessments 2. Enrolled in an ongoing clinical trial, or extension study, expanded access program, or long-term registry of an investigational or recently approved medication 3. Receiving adjuvant and/or dual therapy (e.g., muscle-targeted, NMJ, or symptomatic treatments)
About the study
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.
What is being tested
- Observational (other)
Sponsor: Columbia University · Participants: 45 · Started: Apr 24, 2025
Contact the study team
- Rafael Rodriguez-Torres, DPT · Phone: 3472877093
- Cara Kanner, DPT · Phone: 516-581-8150
Official record on ClinicalTrials.gov — NCT06955897
Locations in the U.S.
| New York | Columbia University Irving Medical Center, New York |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.