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New study tracks how SMA kids change on modern treatments

NCT ID NCT06321965

Knowledge-focused Sponsor: Hospices Civils de Lyon 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 60 children with spinal muscular atrophy (SMA) who are already receiving SMN-restoring therapies like nusinersen or gene therapy. Over two years, researchers will track their muscle function, fatigue, breathing, and thinking skills to describe new disease patterns that have emerged with these treatments. The goal is to better understand how SMA evolves today and to find better ways to monitor children in routine care and future trials.

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.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 60 people

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

Started

Jul 2024

Expected to finish

Jan 2029

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.

Ages

0 to 16 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: * Genetically confirmed infantile or juvenile spinal muscular atrophy * Treated with a therapy that restores SMN protein expression (e.g. nusinersen, risdiplam, onasemnogene abeparvovec) * Aged 0 to 15 years inclusive * Informed consent signed by both parent(s)/legal guardian(s) and patient's assent * Affiliated or beneficiary of a health insurance plan\*. \* for inclusion in France Exclusion Criteria: * Other condition likely to interfere significantly with ASI assessment and clearly unrelated to the disease * Other associated neurological disease * Current pregnancy or breast-feeding (a pregnancy test will also be performed at inclusion). Please note that patients with a specific contraindication to MRI (i.e. metallic foreign body, claustrophobia and other reasons determined by the investigators) will be allowed to participate in the study, but MRI will not be performed.

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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

How to take part

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

  1. The places running it

    8 sites. 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

  • CHRU of Brest

    NOT_YET_RECRUITING

    Brest, 29609, France

  • Department of Pediatrics - Neurology and Infectious Diseases Toulouse University Hospital - Children's Hospital

    NOT_YET_RECRUITING

    Toulouse, 31059, France

  • Hautepierre Hospital - Mother and Child Hospital

    NOT_YET_RECRUITING

    Strasbourg, 67200, France

  • I-Motion Pediatric Clinical Trial Platform Armand Trousseau Hospital

    NOT_YET_RECRUITING

    Paris, 75012, France

  • Marseille University Hospital - Timone Hospital Department of Pediatric Neurology - Specialized Pediatrics and Child Medicine

    NOT_YET_RECRUITING

    Marseille, 13005, France

  • Pediatric Neurology Swynghedauw Hospital

    NOT_YET_RECRUITING

    Lille, 59037, France

  • Pediatric Neurology and Resuscitation Raymond-Poincare Hospital

    NOT_YET_RECRUITING

    Garche, 92380, France

  • Pediatric Rehabilitation Service - L'Escale Mother and Child Hospital

    RECRUITING

    Bron, Rhone, 69500, France

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