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SPINRAZA study tracks movement in adults with spinal muscular atrophy

NCT ID NCT04159987

Disease control Sponsor: Centre Hospitalier Universitaire de Nice 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 2 times

Summary

This study follows 20 adults with spinal muscular atrophy (SMA) type II who are already taking SPINRAZA. Researchers will measure motor function using a 32-point scale over 27 months to see if the drug helps maintain or improve movement. The goal is to better understand how SPINRAZA works in adults with this condition.

What this could mean

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

Active substance
SPINRAZA (nusinersen)
What this could lead to
If successful, this study could confirm that SPINRAZA helps maintain or improve motor function in adults with SMA type II, supporting its continued use.
What could go wrong
This is a small, open-label study with only 20 participants and no placebo group, so results may not apply broadly. It tracks function but does not test a new treatment.

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

20 people

The number who actually took part.

Started

Feb 2020

Expected to finish

Dec 2025

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

18 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: * Adult patients with SMA type II disease who are wheelchair bound, * Must be 18 years or older, * Men or Women with SMA type II disease (age at symptoms onset: \>6 months old and who never acquired the capacity to walk but acquired the ability to sit without support) with genetic confirmed diagnosis of 5q SMA homozygous gene deletion (SMN1 exon 7/8) or mutation or compound heterozygous mutation - performed by PCR amplification and restriction digest of DNA using primers flanking SMN1 and SMN2 exon 7. * MFM 32 score ≥ 19/96 * Lumbar CT scan showing the feasibility of intrathecal injection. * Written informed consent from the subject prior to initiation of any study-mandated procedures * Females of childbearing potential must have a negative pregnancy test at Screening and at Enrollment, must agree to use reliable method of contraception (if sexually active) from screening up to study drug discontinuation plus 180 days. Exclusion Criteria: * Patients with a high risk for thrombocytopenia or hemorrhage or renal diseases: Urine protein, platelet count and coagulation tests will be done prior to intrathecal injection with SPINRAZA® * Patients with high risk of hydrocephalus * Adult patients under guardianship * Pregnant and/or breastfeeding females * Subject has received any investigational therapy or pharmacological treatment for SMA one month prior the beginning of the study * Subject has received gene therapy for SMA

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy spinal muscular atrophy, type II

As listed by the trial registrant

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

Spinal muscular atrophy

Contacts and locations

Locations

  • APHP

    Paris, Île-de-France Region, 94000, France

  • CHRU de Lille

    Lille, Hauts-de-France, 59000, France

  • CHU de Lyon

    Lyon, Auvergne-Rhône-Alpes, 69677, France

  • CHU de Montpellier

    Montpellier, Occitanie, 34090, France

  • CHU de Nice

    Nice, Provence-Alpes-Côte d'Azur Region, 06000, France

  • CHU de Toulouse

    Toulouse, Occitanie, 31059, France

  • Hopital de la Timone - APHM

    Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France

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