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Salanersen for Spinal Muscular Atrophy (SMA)

This study is looking into salanersen (BIIB115) and its effects on movement and safety for people aged 15 to 60 with Spinal Muscular Atrophy (SMA). SMA is a genetic condition where the body doesn't produce enough SMN protein, which is needed for muscles and movement. Researchers want to see how salanersen works in people who are new to SMA treatment or who have previously taken risdiplam. The main goal is to see if salanersen improves movement, measured by a scale called the Hammersmith Functional Motor Scale - Expanded (HFMSE), after 12 months in those who haven't had SMA treatment before. You would need to have genetic confirmation of 5q SMA and clinical signs of the condition to be eligible.

Study design
This is an interventional study with a planned enrollment of 90 participants. It is not specified if it is randomized or blinded.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint is measured at Month 12, suggesting follow-up for at least one year.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07444476

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam

Recruiting
PHASE3Ages 15–60InterventionalTreatment
Biogen
~90 participants
Updated 2026-04-21 on ClinicalTrials.gov
What's tested:Salanersen

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Total Score in Treatment-Naïve Cohort
Measured over At Month 12

Conditions

Spinal Muscular Atrophy

Explore related trials

Spinal Muscular AtrophySMN1 GeneSMN2 Gene

Where it's being run

1 sites across 1 states
Virginia1

Study leadership

  • Medical Director · STUDY_DIRECTOR · Biogen

Who to contact

US Biogen Clinical Trial Center
[email protected]
866-633-4636
Email the study team

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Eligibility criteria

Inclusion

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.

Exclusion

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.

What this trial measures

  • Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Total Score in Treatment-Naïve CohortAt Month 12

    The HFMSE is a tool used to assess motor function in individuals with SMA. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all 33 items, with a maximum score of 66 with higher scores depicting better ability to perform activities.