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Salanersen for Babies with Spinal Muscular Atrophy (SMA)

This study is looking at a medicine called salanersen (BIIB115) in babies who have been diagnosed with Spinal Muscular Atrophy (SMA) through genetic testing, but haven't shown symptoms yet. SMA happens when there isn't enough of a protein called SMN, which is important for muscles and movement. Salanersen works by helping a gene called SMN2 make more of this important protein. Researchers want to see how well salanersen helps with movement and if it is safe. To join, babies must be 42 days old or younger, have a specific genetic diagnosis for SMA, and have 2 or 3 copies of the SMN2 gene. The study aims to see if babies can sit without support or walk alone. The current status of this study is unclear.

Study design
This is an interventional study with a planned enrollment of 30 participants. It is designed to evaluate the effects and safety of salanersen.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed to see if they can sit without support at 12 months, walk alone at 18 months, and achieve motor milestones up to 1825 days (about 5 years).

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

NCT07221669

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)

Recruiting
PHASE3Ages 0–42InterventionalTreatment
Biogen
~30 participants
Updated 2026-08-18 on ClinicalTrials.gov
What's tested:Salanersen

At a glance

Recruiting sites
11 of 11 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Part 1: Percentage of Participants with 2 Survival Motor Neuron 2 (SMN2) Copies Sitting Without Support (for at Least 10 Seconds)
Measured over At Month 12
+2 more outcomes measured

Conditions

Muscular Atrophy, Spinal

Explore related trials

Spinal Muscular AtrophySMN1 GeneSMN2 Gene

Where it's being run

11 sites across 9 states
Brazil2
China2
Illinois1
Texas1
Virginia1
New South Wales1
Rio Grande do Sul1
Henan1

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

≤42 days of age at first dose of salanersen.
Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation.
Two or three copies of the survival motor neuron 2 (SMN2) gene.
Ulnar compound muscle action potential (CMAP) amplitude ≥2 millivolt (mV) at Screening and Day 1 predose.
Body weight ≥3rd percentile for age based on World Health Organization (WHO) Child Growth Standards at the time of informed consent.

Exclusion

Any clinical signs or symptoms at Screening or Day 1 predose that are, in the opinion of the Investigator, strongly suggestive of SMA.
Areflexia on neurologic examination at biceps, knee, or ankle at Screening or Day 1 Predose.
Hypoxemia (oxygen saturation \<96% awake or asleep without any supplemental oxygen or respiratory support, or for altitudes \>1000 meters (m), oxygen saturation of \<92% awake or asleep without any supplemental oxygen or respiratory support).
Diagnosis of neonatal respiratory distress syndrome necessitating surfactant replacement therapy or invasive ventilatory support.
Any reason, anatomical or otherwise (including hematology/coagulation laboratory results), that presents increased risk of complication from the LP procedures or safety assessments.
Any prior treatment with an approved SMA disease-modifying therapy (e.g., nusinersen, onasemnogene abeparvovec-xioi \[OA\], and/or risdiplam), a myostatin inhibitor therapy, or an investigational drug given for the treatment of SMA.

What this trial measures

  • Part 1: Percentage of Participants with 2 Survival Motor Neuron 2 (SMN2) Copies Sitting Without Support (for at Least 10 Seconds)At Month 12
  • Part 1: Percentage of Participants with 3 SMN2 Copies Walking Alone (for at Least 5 Steps)At Month 18
  • Part 2: Percentage of Participants Attaining and Maintaining World Health Organization (WHO) Motor MilestonesUp to Day 1825

    The WHO motor milestones will include six key developmental milestones: sitting without support, standing with assistance, hands-and-knees crawling, walking with assistance, standing alone, and walking alone.