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SMA fatigue mystery: scientists probe exercise limits in 34 patients

NCT ID NCT05518773

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This completed study looked at why people with spinal muscular atrophy (SMA) often feel very tired and have trouble exercising. Researchers tested 34 ambulatory SMA patients who were already on standard treatments (risdiplam or nusinersen) for at least six months. Participants did an exercise test on a special bike to measure peak oxygen uptake, and also had a simple, non-invasive muscle oxygen measurement. The goal was to understand the role of mitochondria in fatigue and exercise intolerance, which could guide future therapies.

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 this study finds clear links between SMA, mitochondrial function, and fatigue, it could point toward new ways to manage tiredness and improve exercise ability in SMA patients.
What could go wrong
This is a small, single-visit observational study with only 34 participants. It does not test any new treatment, so any insights are preliminary and need confirmation in larger, longer studies.

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

34 people

The number who actually took part.

Started

Dec 2022

Finished

Nov 2025

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

The study sample will include 34 ambulatory SMA patients, 17 currently treated with nusinersen, and 17 currently treated with risdiplam.

Ages

8 to 55 years

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: * Genetic confirmation of SMA with laboratory documentation of homozygous deletion of survival motor neuron (SMN1) exon 7; * At least 8 years of age at time of signing Informed Consent Form (or assent) * Children or adults currently receiving treatment, for at least 6 months, with SMN repletion therapy, either with (1) risdiplam, or (2) nusinersen * Able to walk independently at least 25 meters * Able to tread a stationary cycle ergometer. Exclusion Criteria: * Unable to walk 25 meters independently. * Use of investigational medications intended for the treatment of SMA within 30 days prior to study entry. * The presence of any contraindication to exercise according the American College of Sports Medicine (ACSM) criteria.

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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

Contacts and locations

Locations

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.