Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
ConditionSpinal Muscular Atrophy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age12+
SponsorColumbia University
About this trial
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
Qualifiers
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
Disqualifiers
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)
Trial design
Treatments tested in this trial
- Observational
Treatment groups
45 Participants
are divided into 3 treatment groupsSponsors and collaborators
Columbia University
Lead sponsor
Biogen
Collaborator
Source ClinicalTrials.gov