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Can a second treatment boost motor skills in SMA kids after gene therapy?

NCT ID NCT04488133

Disease control Sponsor: Biogen 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study tested the drug nusinersen (Spinraza) in 46 children under 3 years old with spinal muscular atrophy (SMA) who had previously received gene therapy but still had health challenges. The goal was to see if adding nusinersen could improve their muscle and movement abilities. Participants received multiple doses via lumbar puncture over nearly 2 years, and researchers measured motor milestones and safety.

What this could mean

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

Active substance
nusinersen (Spinraza)
What this could lead to
If it works, this could show that adding nusinersen after gene therapy helps improve motor skills in young children with SMA.
What could go wrong
This is a small, completed phase 4 study with no control group, so results may not prove effectiveness. The treatment requires repeated lumbar punctures, which carry risks like headache or infection.

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

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

46 people

The number who actually took part.

Started

Jan 2021

Finished

Oct 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 36 months

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.

Key Inclusion Criteria: For all participants: * Genetic documentation of 5q SMA homozygous gene survival motor neuron 1 (SMN1) deletion or mutation, or compound heterozygous mutation * SMN2 copy number of ≥1 * ≤36 months of age at the time of first Nusinersen dose * Must have previously received onasemnogene abeparvovec per the approved label or local/regional regulations ≥2 months prior to first Nusinersen dose * Must have suboptimal clinical status per the Investigator Additional Criteria for Subgroups A and B: * \<300 days of age at the time of first Nusinersen dose * SMN2 copy number of 2 Additional Criteria for Subgroup A: * SMA symptom onset ≤4 months (120 days) of age * Must have received intravenous (IV) onasemnogene abeparvovec at \>6 weeks to ≤6 months (43 days to 180 days) of age * Must have received IV onasemnogene abeparvovec after SMA symptom onset Additional Criteria for Subgroup B: * Must have received IV onasemnogene abeparvovec at ≤6 weeks (42 days) of age Key Exclusion Criteria: For all participants: * Prior exposure to Nusinersen * Ongoing severe or serious AEs related to onasemnogene abeparvovec * Treatment with an investigational drug, biological agent, or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to study; any prior or current treatment with any survival motor neuron 2 (SMN2)-directed splicing modifier; prior antisense oligonucleotide treatment or cell transplantation; gene therapy for the treatment of SMA other than onasemnogene abeparvovec. Note: treatment with onasemnogene abeparvovec as part of an investigational study is allowed Additional Criteria for Subgroups A and B: * Weight-for-age is below the third percentile, based on WHO Child Growth Standards at the time of receiving onasemnogene abeparvovec. Adjustments for the gestational weight of premature babies enrolled in Subgroups A and B are allowed provided IV onasemnogene abeparvovec was dosed per the approved label or per local/regional regulations. Note: Other protocol defined Inclusion/Exclusion criteria may apply.

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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.

Muscular atrophy, spinal

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital Research Institute

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Philadelphia - Neurology

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of The King's Daughters

    Norfolk, Virginia, 23510, United States

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, Milan, 20133, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 00168, Italy

  • Hospital Sant Joan de Déu

    Esplugues Del Llobregat, Barcelona, 08950, Spain

  • Hospital Universitario La paz

    Madrid, 28046, Spain

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Oregon Health and Science University (OHSU)

    Portland, Oregon, 97239, United States

  • Schneider Children's Medical Center

    Petah Tikva, 4920235, Israel

  • Stanford Neuromuscular Research

    Palo Alto, California, 94304, United States

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • University of Utah

    Salt Lake City, Utah, 84112, United States

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