New yearly shot could help SMA patients move better
NCT ID NCT07444476
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 study tests a drug called salanersen (BIIB115) in people aged 15-60 with spinal muscular atrophy (SMA). The drug is designed to help the body produce more of a protein needed for nerve and muscle function. Participants receive an injection into the spine once a year for up to 5 years. The study includes people who have never been treated for SMA and those who have taken risdiplam. Researchers will measure changes in motor function using movement tests and track any side effects.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- salanersen (BIIB115)
- What this could lead to
- If successful, this could provide a new treatment option for spinal muscular atrophy that helps improve or stabilize motor function with once-yearly dosing.
- What could go wrong
- This is an early-phase open-label study with no placebo group, so results may be less reliable. The drug requires injection into the spine, which carries risks like headache or infection. It may not work for everyone.
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
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 90 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Apr 2026
An estimate. Start dates often move.
- Expected to finish
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Jun 2032
An estimate. End dates often move.
- Lead sponsor
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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
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15 to 60 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: * Participants aged 15 to 60 years, inclusive, at the time of informed consent * Participants with genetic documentation of 5q Spinal Muscular Atrophy (SMA) (homozygous gene deletion or mutation or compound heterozygous mutation). * Participants with clinical signs and symptoms consistent with SMA. * Survival motor neuron 2 (SMN2) copy number ≥ 1. * Participants with baseline Hammersmith Functional Motor Scale - Expanded (HFMSE) total score of ≥ 10 to ≤ 54. * Participants who are able to sit without using support for at least 10 seconds. * Participants with no prior treatment with myostatin inhibitors and a willingness to remain off concurrent myostatin inhibitor therapy for the duration of the study. * Ambulatory and nonambulatory participants: * Ambulatory participants must be able to walk at least 10 meters independently without assistance and are willing and able to complete the 6 Minute Walk Test (6MWT) at Screening. * For participants in the treatment-naïve cohort: * No prior treatment with an approved SMA Disease Modifying Therapy (DMT) or an investigational drug given for the treatment of SMA. * For participants in the risdiplam-treated cohort: * Currently receiving risdiplam treatment and have been on once-daily 5 milligrams (mg) risdiplam treatment for at least 6 months prior to Screening. * Willing to stop risdiplam therapy for the duration of the study. The last dose of risdiplam must be taken the day before the first dose of salanersen. * No prior treatment with nusinersen, onasemnogene abeparvovec-xioi/onasemnogene abeparvovec-brve (OA), other approved DMTs for SMA or investigational drugs given for the treatment of SMA apart from risdiplam. Key Exclusion Criteria: * Respiratory insufficiency at Screening, defined by the medical necessity for invasive or noninvasive ventilation for \> 6 hours during a 24-hour period (except for nocturnal bilevel positive airway pressure). * Medical necessity for a gastric feeding tube, where the majority of nutrition is provided by this route, as assessed by the site Investigator at Screening. * History of brain or spinal cord disease or other contraindications (e.g., severe scoliosis) that would interfere with the lumbar puncture (LP) procedures, Cerebrospinal fluid (CSF) circulation, efficacy assessments, or safety assessments (including a history of hydrocephalus or implanted shunt for CSF drainage), as assessed by the Investigator. * Hospitalization for surgery, a pulmonary event, or nutritional support within 2 months prior to Screening or plans to undergo elective procedures or surgeries at any time after signing the Informed Consent Form (ICF) through the end of the study. Note: If prior scoliosis surgery has been performed, it must be done at least 1 year prior to Screening. * Presence of an active medical issue (e.g., infection, recent fracture) that would make the participant unsuitable for inclusion, as assessed by the Investigator. * Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 90 days or 5 half-lives of the treatment (if known), whichever is longer, prior to Screening. This includes neuromodulation therapy such as spinal cord stimulation. Note: Other protocol-defined inclusion/exclusion criteria will apply.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Childrens Hospital of the Kings Daughter Norfolk
RECRUITINGNorfolk, Virginia, 23507, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- A Baby's fidgets may reveal brain health: study tests early warning signs
- Can a muscle-boosting antibody help people with spinal muscular atrophy over the long haul?
- Can a patient registry unlock the secrets of spinal muscular atrophy?