Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Risdiplam's hidden benefit: fixing Nerve-Muscle links in SMA

NCT ID NCT05219487

Knowledge-focused Sponsor: Bakri Elsheikh 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study looked at 18 adults with spinal muscular atrophy (SMA) who had taken the oral medication risdiplam for at least one year. Researchers measured how well nerves and muscles communicate by using electrical stimulation tests. The goal was to see if risdiplam improves these connections compared to historical data. This was an observational study, meaning participants received their usual treatment, and no new drugs were tested.

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

18 people

The number who actually took part.

Started

Oct 2021

Finished

Dec 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 will enroll genetically confirmed adults with 5 q SMA with electrodiagnostic evidence of NMJ defects treated with risdiplam for a duration of 12 months or longer.

Ages

18 to 70 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: * Signed Informed Consent Form * Age 18-70 years at time of signing Informed Consent Form * Able and willing to provide written informed consent and to comply with the program protocol according to ICH and local regulations * Ability to comply with the study protocol, in the investigator's judgment * Adequately recovered from any acute illness at the time of screening, and considered clinically well enough to participate, in the opinion of the treating physician * Patients with retinopathy of prematurity should have evidence of stable disease * Genetic confirmation of 5q SMA documented on standard genetic tests for the disorder * Treated with risdiplam for a duration of 12 months or longer Exclusion Criteria: * Treatment with an investigational therapy within 180 days prior to initiation of study drug * History of established diagnosis of neuromuscular junction disorder * Co-morbid conditions that preclude travel or testing * Patients who are, in the investigator's opinion, mentally or legally incapacitated to provide an informed consent

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Spinal muscular atrophy are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

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

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210, United States

More trials for these conditions

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