Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA

Part of paid clinical trials in New York, New York.

Sponsor
Columbia University
Study ID
NCT06955897
Status
Recruiting
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Conditions

  • Spinal Muscular Atrophy

Eligibility Criteria

Sex
ALL
Age
12 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Observational — OTHER
    Observational

Study Details

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.

Key Dates

Start date
Apr 24, 2025
Status verified
May 2026
Primary completion
Dec 7, 2026
Completion
Feb 26, 2027

Study Design

Enrollment
45 participants (estimated)

Arms

  • Arm: Walkers
    Participants with SMA who are able to walk without assistance.
  • Arm: Sitters
    Participants with SMA who are unable to walk without assistance, but are able to sit independently.
  • Arm: Non-sitters
    Participants with SMA who are unable to sit without assistance.

Primary Outcome Measure

SMA EFFORT [ Time Frame: Baseline, 1 week, 1 month ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Columbia University Irving Medical CenterNew YorkNew York10032
Jacqueline Montes, PT EdD
[email protected]
2123058916
Rafael Rodriguez-Torres, DPT
[email protected]
347-287-7093

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By research site
Columbia University Irving Medical Center· New York, NY

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