A Phase 3, Randomized, Double-Blinded Study to Evaluate the Safety and Efficacy of Salanersen (BIIB115) After Onasemnogene Abeparvovec Treatment in Infants with Genetically Diagnosed Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a rare genetic condition that weakens muscles and affects how babies move. Many infants now receive gene therapy to slow down or halt the condition. This clinical trial is testing a new medicine called salanersen (also known as BIIB115) to see if it can provide additional help. The trial is for babies who have already been treated with an SMA gene therapy called onasemnogene abeparvovec. Doctors want to find out whether salanersen is safe and if it helps babies achieve important physical milestones, such as rolling, sitting without support, crawling, and walking. Over the course of the study, doctors will track the children's motor skills, check nerve signals in their muscles, and run simple blood tests to look for signs of nerve health. The study monitors participants for up to five years.
At a glance
What is this study about?
Spinal muscular atrophy (SMA) is a condition caused by a missing or faulty gene. Without this gene, the body cannot make enough of a protein that keeps motor nerves healthy. Over time, this leads to muscle weakness, making everyday movements like kicking, crawling, and sitting difficult. While gene therapy has dramatically changed how SMA is treated, doctors want to find out if combining treatments can help babies gain even more strength.
This study focuses on a new medicine called salanersen (BIIB115). It is designed to work alongside previous gene therapy to support nerve cells and muscle function. Researchers want to learn how well the body tolerates salanersen and whether it offers meaningful improvements in movement beyond what gene therapy achieves on its own.
Throughout the study, specialist doctors will carefully monitor each child. They will look at whether children reach key developmental milestones, such as holding their head up, sitting without help, crawling, and walking. They will also use non-invasive tests to measure nerve-to-muscle signals and test small blood samples to check for signs that nerves are protected from damage.
Because SMA is a lifelong condition, studies like this are vital. They help doctors understand if giving additional targeted treatments early in life can give children the best possible chance to move more easily and live active lives.
Key takeaways
- Tests a new medicine, salanersen, for babies with spinal muscular atrophy.
- Open to children who have already received onasemnogene abeparvovec gene therapy.
- Aims to improve muscle strength, movement, and physical developmental milestones.
- Tracks safety, nerve health, and progress for up to five years.
- Participation is entirely voluntary and will not impact standard NHS healthcare.
Who may be eligible?
This trial is designed specifically for babies and young children who have been diagnosed with spinal muscular atrophy (SMA) through genetic testing. Both boys and girls can take part.
To be eligible, infants must have already received a specific gene therapy treatment known as onasemnogene abeparvovec. This study is testing how well salanersen works after that gene therapy has been given.
The trial doctors will review each child's complete medical history, previous treatments, and overall health to make sure that joining the study is safe and appropriate for them.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- My child has a confirmed genetic diagnosis of spinal muscular atrophy (SMA).
- My child has already received the gene therapy onasemnogene abeparvovec.
- My child is an infant or young child.
- Our family is able to attend regular clinic appointments for checks and assessments over several years.
What does participation involve?
Taking part involves regular visits to a specialist hospital or clinical trial site. Because this is a blinded study, neither you nor the study team will know right away whether your child receives salanersen or a comparison treatment, which ensures the results are completely fair. During clinic visits, doctors will give the study medicine and check your baby's general health, growth, and developmental progress. Visits will include physical assessments to see how your baby rolls, sits, or crawls, as well as tests called CMAP, which use gentle electrical patches on the skin to check how nerves talk to muscles. Doctors will also take blood samples to measure nerve health. The study is split into two parts: an initial one-year phase (Part A) to check early safety and progress, followed by longer-term follow-up (Part B) that monitors your child's health and development for up to five years in total.
Potential risks and benefits
Locations (10)
- —UnverifiedPortugal
- —UnverifiedItaly
- —UnverifiedBelgium
- —UnverifiedCroatia
- —UnverifiedHungary
- —UnverifiedGermany
- —UnverifiedIreland
- —UnverifiedFrance
- —UnverifiedNorway
- —UnverifiedPoland
Common questions
What is salanersen?
Salanersen (BIIB115) is an experimental medicine being tested to see if it can boost nerve and muscle function in babies with spinal muscular atrophy.
Does my child have to stop their standard care to join?
Children in this study must have already had onasemnogene abeparvovec gene therapy. Your study doctor will guide you on how standard care and supportive therapies fit with the trial.
What does 'double-blind' mean?
It means neither you nor the doctors will know whether your child is receiving the active medicine or a comparison treatment until the study code is opened, which prevents bias in the results.
How long will our family be in the study?
The study has two parts: an initial one-year evaluation followed by a longer monitoring period lasting up to five years in total.
Will taking part cost anything?
Clinical trial medicines and all study-related visits and tests are provided without charge, and travel expenses are often reimbursed by the study.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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