Actively Recruiting
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
Led by Biogen · Updated on 2026-04-21
90
Participants Needed
1
Research Sites
324 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in individuals with SMA who are between the ages of 15 and 60 years old. In most people living with SMA, changes to or a lack of a gene called survival motor neuron 1 (SMN1) - often referred to as gene mutations or variants - affect how this gene works. As a result, their bodies produce less SMN protein. Without enough of this protein, motor neurons and muscles cannot work properly. There is a similar gene called SMN2 that produces SMN protein, but it usually does not produce enough SMN protein on its own to make up for the changes in the SMN1 gene. Salanersen is a drug designed to help the SMN2 gene to make more working SMN protein. In this study, there will be 2 groups of participants: a group who has never received treatment for SMA before joining this study, and a group who has been treated with risdiplam, an approved drug for SMA . Those participants must not have received any other SMA treatments before and will need to stop their risdiplam treatment for the duration of the study. The main goal of this study is to learn more about how salanersen affects the participants' motor function. Researchers will use different tests and questionnaires to learn if motor function is changing over the study duration. The main question researchers want to answer in this study is: • For the group who has never been treated for SMA, how much do scores on the HFMSE movement test change at 12 months compared to the beginning of the study? The Hammersmith Functional Motor Scale - Expanded (HFMSE) has 33 activities that are scored which include sitting, lying down, walking, jumping, and more. Researchers will also learn more about: * The effects on participants' motor function and how well their nerves and muscles function. * The effects on participants' overall sense of change and how they perform daily activities. * How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug. * How much salanersen gets into the fluid surrounding the brain and spinal cord. * How much salanersen gets into the blood. This study will be done as follows: * First, participants will be screened to check if they can join the study. The screening period may be up to 4 weeks. * This is an "open-label" study. This is a study in which the participants, study doctor, and site staff will know that participants are receiving salanersen. * All participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord. * Participants will receive salanersen once every year for a total of 5 times throughout the study. * Including screening, participants will have 17 study visits and 9 telephone calls during this study, which will last up to 61 months in total.
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants aged 15 to 60 years at the time of consent
- Genetic confirmation of 5q Spinal Muscular Atrophy (SMA) with homozygous deletion or mutation
- Clinical signs and symptoms consistent with SMA
- SMN2 gene copy number of 1 or more
- Baseline Hammersmith Functional Motor Scale - Expanded (HFMSE) score between 10 and 54
- Ability to sit without support for at least 10 seconds
- No prior treatment with myostatin inhibitors and willingness to avoid them during the study
- Ambulatory participants must be able to walk at least 10 meters independently and complete a 6 Minute Walk Test at screening
- For treatment-naive participants: no prior use of approved SMA disease modifying therapies or investigational SMA drugs
- For risdiplam-treated participants: currently on daily 5 mg risdiplam for at least 6 months before screening, willing to stop risdiplam for the study duration, and no prior treatment with other approved SMA therapies or investigational drugs except risdiplam
You will not qualify if you...
- Respiratory insufficiency requiring invasive or noninvasive ventilation for more than 6 hours a day (except nocturnal bilevel positive airway pressure)
- Need for a gastric feeding tube providing most nutrition as assessed at screening
- History of brain or spinal cord disease or conditions interfering with lumbar puncture or cerebrospinal fluid circulation
- Recent hospitalization for surgery, pulmonary event, or nutritional support within 2 months before screening
- Planned elective surgeries from consent through study end
- Scoliosis surgery within 1 year prior to screening
- Active medical issues like infection or recent fracture making participation unsuitable
- Current or planned enrollment in another investigational clinical study within 90 days or 5 half-lives of prior treatment, including neuromodulation therapy
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Childrens Hospital of the Kings Daughter Norfolk
Norfolk, Virginia, United States, 23507
Actively Recruiting
Research Team
U
US Biogen Clinical Trial Center
CONTACT
G
Global Biogen Clinical Trial Center
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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