Fondazione I.R.C.C.S. Istituto Neurologico Besta
Milan, Italy
Not recruitingRare disease
Study of Nusinersen for Patients with Spinal Muscular Atrophy Previously Treated with Onasemnogene Abeparvovec
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What is this trial about?
A plain-language summary of the goals, design and what participants do
This clinical trial is focused on studying a condition known as Spinal Muscular Atrophy (SMA), a genetic disorder that affects the muscles used for movement. The study involves a treatment called Nusinersen, which is also known by its code name BIIB058. Nusinersen is administered as a solution for injection directly into the spinal canal, a method known as intrathecal use. The purpose of the study is to evaluate the effects of Nusinersen in individuals with SMA who have previously been treated with another medication called Onasemnogene Abeparvovec.
Participants in this study will receive Nusinersen over a period of time, and their progress will be monitored to assess the treatment's impact on their condition. The study will track various health indicators, including motor skills and any potential side effects. The goal is to understand how Nusinersen can help improve the clinical outcomes for those with SMA who have already received Onasemnogene Abeparvovec.
Throughout the study, participants will undergo regular assessments to monitor changes in their health status. These assessments will include evaluations of motor milestones, which are key developmental skills, as well as checks for any adverse events or changes in vital signs. The study aims to provide valuable insights into the effectiveness of Nusinersen for individuals with SMA, contributing to better treatment strategies for this condition.
The research process
The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
11 criteria
- Must have a genetic confirmation of **5q Spinal Muscular Atrophy (SMA)**, which means a specific change or deletion in the **SMN1 gene**.
- Must have at least one copy of the **SMN2 gene**.
- Must be 36 months old or younger at the time of the first dose of **Nusinersen**, a medication used to treat SMA.
- Must have previously received a treatment called **onasemnogene abeparvovec** at least 2 months before the first dose of Nusinersen.
- Must have a less than ideal health condition as determined by the study doctor.
- For certain groups, must be younger than 300 days at the time of the first Nusinersen dose.
- For certain groups, must have two copies of the **SMN2 gene**.
- For a specific group, SMA symptoms must have started at 4 months of age or younger.
- For a specific group, must have received **intravenous (IV) onasemnogene abeparvovec** treatment after SMA symptoms began.
- For another specific group, must have received IV onasemnogene abeparvovec treatment at 6 weeks of age or younger.
- Both males and females can participate.
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Verified sites
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Verified Verified Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, ItalyResponsive Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Madrid, Spain
Trial locations
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Investigated drugs
Nusinersen
is a medication used to treat spinal muscular atrophy (SMA). It works by helping to increase the production of a protein that is essential for the health and function of motor neurons, which are the nerve cells that control muscle movement. This medication is administered through an injection into the spinal fluid, allowing it to reach the central nervous system directly.
Onasemnogene Abeparvovec
is a gene therapy used to treat spinal muscular atrophy (SMA). It delivers a copy of the gene that is missing or not working properly in people with SMA. This therapy aims to improve muscle movement and strength by addressing the genetic cause of the disease. It is given as a one-time intravenous infusion.
What is already known about the treatment
Nusinersen – Nusinersen is administered through an injection into the spinal canal, a method known as intrathecal injection. It is currently approved and widely used in the treatment of spinal muscular atrophy (SMA), with extensive documentation in medical literature supporting its efficacy. The main therapeutic indication for nusinersen is to improve motor function in individuals with SMA. At the molecular level, nusinersen works by increasing the production of a protein called survival motor neuron (SMN), which is crucial for the health of motor neurons. It is classified as an antisense oligonucleotide, a type of medication that targets specific RNA molecules to modify protein production.
Investigated diseases
Spinal Muscular Atrophy – Spinal Muscular Atrophy (SMA) is a genetic disorder characterized by the progressive loss of motor neurons, which are nerve cells in the spinal cord that control muscle movement. This leads to muscle weakness and atrophy, primarily affecting the muscles closest to the center of the body, such as those in the shoulders, hips, and back. SMA is caused by mutations in the SMN1 gene, which is responsible for producing a protein essential for motor neuron survival. The severity of the disease can vary, with some individuals experiencing symptoms in infancy, while others may not show signs until later in childhood or adulthood. As the disease progresses, individuals may experience difficulties with movement, swallowing, and breathing. The progression rate and severity can differ significantly among individuals with SMA.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2023-505640-18-00Protocol code232SM404Estimated enrolment46 patientsSponsorBiogen Idec Research Limited
sourced from the EU Clinical Trials Register and site verification
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