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New study aims to measure fatigue in SMA patients on nusinersen

NCT ID NCT06955897

Knowledge-focused Sponsor: Columbia University Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study looks at how tired people with spinal muscular atrophy (SMA) feel during daily activities while taking the drug nusinersen. Researchers will use a new questionnaire called the SMA EFFORT to measure this fatigue. The goal is to better understand fatigue in SMA and see if it changes before and after nusinersen doses. About 45 adolescents and adults who have been on stable nusinersen for at least 6 months will take part.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If successful, this study could provide a better way to measure fatigue in SMA patients, helping doctors tailor treatments and improve quality of life.
What could go wrong
This is an observational study with only 45 participants, so results may not apply to all SMA patients. The new measurement tool may not capture fatigue accurately.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 45 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2025

Expected to finish

Feb 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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)

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Conditions

The condition(s) this trial relates to.

Fatigue Motor Activity neuromuscular disease spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Spinal muscular atrophy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Columbia University Irving Medical Center

    RECRUITING

    New York, New York, 10032, United States

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