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Hunting molecular clues to predict how SMA unfolds

NCT ID NCT07883655

Diagnosis Sponsor: Istituto Giannina Gaslini 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Oct 20, 2026 · Last updated Oct 21, 2026 · Updated 1 time

Summary

Spinal muscular atrophy (SMA) is an inherited nerve and muscle disease caused by faults in the SMN1 gene. Doctors struggle to predict how quickly it will progress or who will respond best to treatment. This study collects blood and other biological samples from people with SMA and from healthy controls, then uses advanced molecular profiling to search for biomarkers. Researchers aim to find markers that separate SMA from healthy controls, flag disease subtypes, and forecast progression in newborns.

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 it works, this could give doctors better tools to predict how SMA will progress and to tell early whether a treatment is helping.
What could go wrong
This is a biomarker discovery study, so it may not find markers that hold up in larger groups or that change how doctors care for patients. It offers no direct treatment to participants.

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 200 people

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

Started

Feb 2025

Expected to finish

Dec 2026

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

The study will include three groups: 1. Patients with a genetically confirmed diagnosis of SMA types 1, 2, or 3. 2. Newborns diagnosed with SMA through newborn screening, with genetic confirmation. 3. Healthy age-matched controls with no personal or family history of neuromuscular diseases.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

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 diagnosis of SMA, with homozygous deletion or mutation of the SMN1 gene, for symptomatic patients. * Diagnosis of SMA through newborn screening, with genetic confirmation, for newborns. * Ability of parents/legal guardians to provide informed consent for minor patients. * Willingness to undergo the study procedures, including blood sampling and collection of biological specimens. Exclusion Criteria: * Presence of other neuromuscular or neurological diseases that could influence the clinical phenotype and biomarker profile. * Participation in other clinical trials involving investigational medicinal products for SMA within the 6 months prior to enrolment. * Presence of medical or psychological conditions that could compromise patient compliance or study safety. * Refusal to participate in the study or to provide informed consent.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy spinal muscular atrophy, type IV

As listed by the trial registrant

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

Spinal muscular atrophy (SMA)

How to take part

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

  1. The places running it

    3 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

  • Fondazione Ca' Granda Ospedale Maggiore Policlinico

    RECRUITING

    Milan, Italy

  • IRCCS Istituto Giannina Gaslini

    RECRUITING

    Genova, Italy

  • Istituto di Bioscienze e Biorisorse IBBR

    RECRUITING

    Naples, Italy

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