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

NCT ID NCT05067790

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

45 people

The number who actually took part.

Started

Jan 2022

Expected to finish

Sep 2027

An estimate. End dates often move.

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

15 to 50 years

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: * Genetic documentation of 5q SMA homozygous survival motor neuron-1 (SMN1) gene deletion or mutation or compound heterozygous mutation. * Diagnosis of later-onset SMA with symptom onset at age \>6 months. * Aged ≥15 to ≤50 years at the time of informed consent * Body weight \>20 kg. * Received oral risdiplam per the approved label or per the managed access program as follows 1. Nusinersen-naive participants must have had prior treatment with risdiplam for ≥6 months before enrollment. 2. Nusinersen-experienced participants must have stopped nusinersen for ≥16 months and must have been on risdiplam for ≥12 months before enrollment. * Able to perform the age-appropriate functional assessments in the study. * RULM entry item A score ≥3. * RULM total score ≥5 and ≤30 at Screening. * Nonambulatory, defined as not able to walk 15 feet (4.57 meters) independently without support. * Willing to stop risdiplam treatment. * Willing and able to start treatment with HD nusinersen. Key Exclusion Criteria: * Any major illness within 1 month before the screening examination or within 1 week prior to Screening and up to first dose administration. * Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the Screening Period. * Presence of an implanted shunt for the drainage of CSF or of an implanted central nervous system catheter. * Permanent tracheostomy or permanent ventilation at Screening. * The medical necessity, as defined by the Investigator, for noninvasive ventilation such as bilevel positive airway pressure or continuous positive airway pressure outside of regular sleep hours for any reason other than proactive SMA management, at Screening. * History of bacterial meningitis, viral encephalitis, or hydrocephalus. * Ongoing medical condition that according to the Investigator would interfere with the conduct and assessments of the study. An example is a medical disability (e.g., wasting or cachexia, severe anemia, and respiratory parameters) that would interfere with the assessment of safety or would compromise the ability of the participant to undergo study procedures. * Participants who are pregnant or currently breastfeeding and those intending to become pregnant during the study. * Treatment with an investigational drug, biological agent, or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to Screening or anytime during the study; any prior or current treatment with gene therapy for the treatment of SMA. 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.

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

More trials for these conditions

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