NCT06955897Recruiting
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
Characterizing Perceived Physical Fatigability in Nusinersen-treated Adolescents and Adults: The SMA EFFORT
Source: ClinicalTrials.gov · Last updated 14 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 45 (estimated)
- First submitted
- 25 Apr 2025
- Start date
- 24 Apr 2025
- Primary completion date
- 6 Sept 2027
- Lead sponsor
- Columbia University
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT06955897
What you commit to
- Study type: Observational
- Planned participants: 45
- From 24 Apr 2025 to the primary completion date, 6 Sept 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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)
Who the study is looking for
- 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
Age in the registry: 12 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Columbia University Irving Medical Center Coordinator of this site? Confirm its status | New York | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.