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

NCT ID NCT04488133

Completed Disease control Sponsor: Biogen Source: ClinicalTrials.gov ↗

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.

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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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