NCT06955897 Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
| NCT ID | NCT06955897 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Columbia University |
| Condition | Spinal Muscular Atrophy |
| Study Type | OBSERVATIONAL |
| Enrollment | 45 participants |
| Start Date | 2025-04-24 |
| Primary Completion | 2026-12-07 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 45 participants in total. It began in 2025-04-24 with a primary completion date of 2026-12-07.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
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)
Contact & Investigator
Jacqueline Montes, Pt, EdD
PRINCIPAL INVESTIGATOR
Columbia University
Frequently Asked Questions
Who can join the NCT06955897 clinical trial?
This trial is open to participants of all sexes, aged 12 Years or older, studying Spinal Muscular Atrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06955897 currently recruiting?
Yes, NCT06955897 is actively recruiting participants. Contact the research team at rsr2157@cumc.columbia.edu for enrollment information.
Where is the NCT06955897 trial being conducted?
This trial is being conducted at New York, United States.
Who is sponsoring the NCT06955897 clinical trial?
NCT06955897 is sponsored by Columbia University. The principal investigator is Jacqueline Montes, Pt, EdD at Columbia University. The trial plans to enroll 45 participants.