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Higher dose of spinraza tested in SMA patients who already tried risdiplam

NCT ID NCT05067790

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

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a higher dose of nusinersen (Spinraza) in 45 teenagers and adults with spinal muscular atrophy (SMA) who cannot walk and have already taken risdiplam. Participants receive two 50 mg loading doses two weeks apart, then 28 mg every four months for about two years, with an option to continue. The main goal is to see if this higher dose improves arm and hand function, measured by the Revised Upper Limb Module test.

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 successful, this could offer a more effective treatment option for SMA patients who have already tried risdiplam, potentially improving arm and hand function.
What could go wrong
This is a small, early-phase study (45 participants) and results may not apply to all SMA patients. Higher doses may increase side effects, and the drug is given via lumbar puncture, which carries 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.

SPINAL MUSCULAR ATROPHY

Contacts and locations

Locations

  • Barrow Neurological Institute

    Phoenix, Arizona, 85013, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115-5724, United States

  • Columbia University

    New York, New York, 10032, United States

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 168, Italy

  • Fondazione Serena Onlus - Centro Clinico Nemo

    Milan, 20162, Italy

  • Klinikum rechts der Isar der TU Muenchen

    Munich, Bavaria, 81675, Germany

  • Memorial Healthcare

    Owosso, Michigan, 48867, United States

  • NHO Osaka Toneyama Medical Center

    Toyonaka-shi, Osaka, 560-8552, Japan

  • Neurology Rare Disease Center

    Denton, Texas, 76208, United States

  • Ospedale S G Battista Molinette

    Torino, 10126, Italy

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30329, United States

  • Semmelweis Egyetem

    Budapest, 1085, Hungary

  • Stanford Neuroscience Health Center

    Palo Alto, California, 94304, United States

  • The University of Texas Health Science Center at Houston

    Houston, Texas, 77030, United States

  • Universitaetsklinikum Essen

    Essen, North Rhine-Westphalia, 45122, Germany

  • Universitaetsklinikum Heidelberg

    Heidelberg, Baden-Wurttemberg, 69120, Germany

  • Universitaetsklinikum Ulm

    Ulm, Baden-Wurttemberg, 89081, Germany

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Washington Medical Center

    Seattle, Washington, 98195, United States

  • Wake Forest University - School of Medicine - Central

    Winston-Salem, North Carolina, 27157, United States

  • Yokohama City University Hospital

    Yokohama, Kanagawa, 236-0004, Japan

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