Study of Nusinersen for Patients with Spinal Muscular Atrophy Previously Treated with Onasemnogene Abeparvovec

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What is this study about?

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.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying genetic documentation of spinal muscular atrophy (SMA) and previous treatment with onasemnogene abeparvovec.

The assessment ensures that participants meet specific criteria, such as age and clinical status, to proceed with the trial.

2 first dose administration

The first dose of nusinersen is administered. Nusinersen is given as a solution for injection through a procedure called intrathecal use, which involves delivering the medication directly into the spinal canal.

This step is crucial for initiating the treatment process and is conducted under medical supervision.

3 follow-up doses

Subsequent doses of nusinersen are administered at regular intervals. The frequency and schedule of these doses are determined by the study protocol and are essential for maintaining the treatment’s effectiveness.

Participants are monitored for any changes in their condition and for any potential side effects.

4 regular assessments

Throughout the trial, regular assessments are conducted to evaluate the participant’s progress. These assessments include measuring motor milestones and other clinical outcomes.

The assessments help in understanding the impact of nusinersen on the participant’s condition over time.

5 monitoring for adverse events

Participants are closely monitored for any adverse events or changes in health status. This includes tracking any side effects or serious adverse events that may occur.

Monitoring is an ongoing process to ensure participant safety and to address any issues promptly.

6 final evaluation

At the end of the study period, a final evaluation is conducted. This includes a comprehensive review of the participant’s health and the overall outcomes of the treatment.

The final evaluation helps in determining the long-term effects of nusinersen and its efficacy in treating spinal muscular atrophy.

Who Can Join the Study?

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.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Ugnzesdesy Mocwtui Cohzoc Hkjeemhlfbmsnrobp	Hamburg	Germany
Fnwzaatea Pzah Ls Iytcynlkhfnfi Boxkogvek Dyk Hbehalur Ukjglhwgqcdqm Li Pue	Madrid	Spain

Trial status

Country	Status	Recruitment Start
Germany	Not recruiting	02.09.2021
Italy	Not recruiting	02.09.2021
Spain	Not recruiting	02.09.2021

Trial locations

Investigated drugs:

Nusinersen

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.

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 ID:

2023-505640-18-00

Protocol code:

232SM404

NCT ID:

NCT04488133

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study of Nusinersen for Patients with Spinal Muscular Atrophy Previously Treated with Onasemnogene Abeparvovec

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