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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

This trial does not accept healthy volunteers

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 population

12 years and older, with genetic diagnosis of all types of 5qSMA, treated with nusinersen (12 or 28 mg) for at least 6 months, will be included.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Observational

    Other intervention

    Observational

Treatment groups

45 Participants
are divided into 3 treatment groups
Group A: Walkers1 intervention
Group B: Sitters1 intervention
Group C: Non-sitters1 intervention

Trial outcomes

Primary outcomes

1

SMA EFFORT

The SMA EFFORT is a perceived physical fatigability patient-reported outcome measure (PROM) that provides a percent (PPF%) total score to each participant based on their participation over the past 30 days.

Time frame
Baseline, 1 week, 1 month

Secondary outcomes

1

Revised Upper Limb Module (RULM)

The RULM is used to assess motor function of the shoulder, mid-level elbow, wrist, and hand.

Time frame
Baseline
2

Hammersmith Functional Motor Scale Expanded (HFMSE)

The HFMSE is a 33-item scale designed to assess gross motor function in sitters and walkers with SMA.

Time frame
Baseline
3

Six-Minute Walk Test (6MWT)

The 6MWT is an objective evaluation of functional exercise capacity which measures the maximum distance a person can walk in six minutes over a 25-meter linear course.

Time frame
Baseline
4

Six-Minute Mastication Test (6MMT)

The 6MMT is a measure of chewing endurance in which the individual is asked to continuously chew on a chewing tube for a period of six minutes.

Time frame
Baseline

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Columbia University

Lead sponsor

Biogen

Collaborator

Source ClinicalTrials.gov
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