Actively Recruiting
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Led by Columbia University · Updated on 2026-05-14
45
Participants Needed
1
Research Sites
96 weeks
Total Duration
On this page
Sponsors
C
Columbia University
Lead Sponsor
B
Biogen
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Who Can Participate
Eligibility Criteria
You may qualify if you...
- All types of 5qSMA with 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
You will not qualify if you...
- Injury or surgery within the previous 3 months affecting ability to perform function or fatigability assessments
- Enrolled in an ongoing clinical trial, extension study, expanded access program, or long-term registry of investigational or recently approved medication
- Receiving adjuvant and/or dual therapy such as muscle-targeted, NMJ, or symptomatic treatments
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
Research Team
R
Rafael Rodriguez-Torres, DPT
CONTACT
C
Cara Kanner, DPT
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
3
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here