Actively Recruiting
An Open-Label Study to Evaluate the Effects and Safety of Salanersen (BIIB115) in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy, Including Those New to Treatment or Previously Treated With Risdiplam
Led by Biogen · Updated on 2026-04-21
90
Participants Needed
1
Research Sites
208 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effects and safety of BIIB115, also called salanersen, in people aged 15 to 60 years with spinal muscular atrophy (SMA). This condition is caused by changes in the SMN1 gene, leading to low levels of SMN protein, which affects motor neurons and muscles. Salanersen aims to increase SMN protein production from the SMN2 gene. The study includes two groups: those new to SMA treatment and those previously treated with risdiplam, who must stop risdiplam during the study. Participants will receive salanersen by intrathecal injection into the fluid around the brain and spinal cord once every 12 months, for a total of five doses over the study period. This open-label study involves 17 visits and 9 phone calls spanning up to 61 months. Both treatment-naïve and risdiplam-treated groups will receive the same dosing schedule. During the study, researchers will assess changes in motor function using tests like the Hammersmith Functional Motor Scale - Expanded (HFMSE) and other measures of nerve and muscle performance. They will monitor safety, including adverse events, and measure salanersen levels in cerebrospinal fluid and blood. Participants will be closely followed to understand how salanersen affects their ability to perform daily activities over the course of the trial.
CONDITIONS
Brief 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...
- Aged 15 to 60 years at the time of consent
- Genetic confirmation of 5q Spinal Muscular Atrophy (SMA)
- 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 the 6-Minute Walk Test at screening
- For treatment-naïve group: no prior SMA disease-modifying therapies or investigational SMA drugs
- For risdiplam-treated group: currently on daily 5 mg risdiplam for at least 6 months, willing to stop risdiplam for the study, and no prior treatment with other SMA therapies except risdiplam
You will not qualify if you...
- Respiratory insufficiency requiring invasive or noninvasive ventilation over 6 hours in 24 hours (except at night)
- Need for a feeding tube providing most nutrition
- Brain or spinal cord disease or conditions interfering with lumbar puncture or assessments
- Hospitalization for surgery, pulmonary event, or nutritional support within 2 months before screening
- Planned elective surgeries during the study period
- Active medical issues like infection or recent fracture making the participant unsuitable
- Participation in another interventional clinical study with investigational or approved therapies within 90 days or 5 half-lives before screening
- History of scoliosis surgery less than 1 year before screening
- Use of neuromodulation therapies such as spinal cord stimulation before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 5 years
Participants receive salanersen 80 mg by intrathecal lumbar puncture every 12 months for a total of five doses.
5 treatment visits (in-person) spaced 12 months apart
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
G
Global Biogen Clinical Trial Center
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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