Nusinersen for SMA
(RESPOND Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores how nusinersen affects muscle and movement abilities in young children with spinal muscular atrophy (SMA) who continue to struggle after previous gene therapy. Researchers aim to observe improvements in motor functions, such as sitting and crawling, while monitoring safety and any side effects. This study suits children under 3 years old with SMA who have had limited success with the gene therapy onasemnogene abeparvovec. Participants will receive nusinersen through a lumbar puncture (an injection into the lower back) over several months. Researchers will closely monitor safety and movement abilities throughout the study. As a Phase 4 trial, this research helps determine how an already FDA-approved and effective treatment can benefit more patients.
Will I have to stop taking my current medications?
The trial protocol does not specify if you need to stop taking your current medications. However, you cannot participate if you have been treated with certain investigational drugs or treatments for SMA other than onasemnogene abeparvovec within 30 days before the study.
What is the safety track record for nusinersen?
Research has shown that nusinersen is generally safe for people with spinal muscular atrophy (SMA). In earlier studies, about 58% of those treated experienced higher protein levels in their urine, but no major safety issues were directly linked to nusinersen.
Reported side effects include fever, cough, pneumonia, and upper respiratory infections. Despite these, the treatment is widely used and approved in over 71 countries for people of all ages with SMA, indicating its safety.
Overall, while some side effects exist, past research has not identified serious safety problems with nusinersen.12345Why are researchers enthusiastic about this study treatment?
Researchers are excited about Nusinersen for treating spinal muscular atrophy (SMA) because it offers a targeted approach by boosting the production of the survival motor neuron (SMN) protein, which is crucial for muscle function. Unlike other treatments that might focus on managing symptoms or providing general support, Nusinersen is administered directly into the spinal fluid via intrathecal injection, allowing it to act directly on the central nervous system where it's needed most. This targeted delivery method is designed to help improve or stabilize motor function in patients with SMA, offering a potentially significant improvement over conventional therapies.
What is the effectiveness track record for nusinersen in treating SMA?
Studies have shown that nusinersen can improve movement in people with spinal muscular atrophy (SMA). Research indicates that patients treated with nusinersen, also known as SPINRAZA, have experienced lasting improvements in movement, regardless of age or type of SMA. Specifically, early results from the RESPOND study showed that many participants who had already received the gene therapy Zolgensma saw further improvement in motor skills when treated with nusinersen. With a well-established safety record and evidence supporting its effectiveness over many years, nusinersen offers hope for enhancing muscle and movement abilities in children with SMA.678910
Who Is on the Research Team?
Medical Director
Principal Investigator
Biogen
Are You a Good Fit for This Trial?
This trial is for children under 36 months with Spinal Muscular Atrophy who have a specific genetic mutation and at least one copy of the SMN2 gene. They must have previously received onasemnogene abeparvovec, not been exposed to Nusinersen before, and can't be suffering from severe side effects related to onasemnogene abeparvovec.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive 4 initial doses of 12 mg of nusinersen on Days 1, 15, 29, and 64, followed by maintenance doses every 4 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Nusinersen
Nusinersen is already approved in United States, European Union for the following indications:
- Spinal muscular atrophy in pediatric and adult patients
- Spinal muscular atrophy
Find a Clinic Near You
Who Is Running the Clinical Trial?
Biogen
Lead Sponsor
Daniel Quirk
Biogen
Chief Medical Officer
MD
Christopher A. Viehbacher
Biogen
Chief Executive Officer since 2022
Graduated from Queen's University, Kingston, Ontario, Canada