Nusinersen for Spinal Muscular Atrophy
(NURTURE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines how well the medicine Nusinersen works for babies with spinal muscular atrophy (SMA) before symptoms appear. The main goal is to determine if Nusinersen can delay or prevent serious breathing problems or death. Eligible participants are babies under six weeks old with a genetic diagnosis of SMA but no symptoms. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group.
Will I have to stop taking my current medications?
The trial information does not specify whether participants must stop taking their current medications. It's best to discuss this with the study team or your doctor.
Is there any evidence suggesting that Nusinersen is likely to be safe for humans?
Research has shown that nusinersen has been studied for its safety in treating spinal muscular atrophy (SMA). In long-term studies, patients generally handled the treatment well. Common side effects include headache, back pain, and nausea, but these were not severe for most people.
Nusinersen is approved in over 71 countries, suggesting it is safe for treating SMA. This approval indicates widespread use, offering more information on its safety. However, like any treatment, risks exist, so discussing any concerns with the trial team or your doctor is important.12345Why do researchers think this study treatment might be promising for SMA?
Unlike the standard treatments for Spinal Muscular Atrophy (SMA) that often target symptoms or require frequent administration, Nusinersen works by enhancing the production of a critical protein called SMN, which is vital for motor neuron survival. This drug is administered directly into the spinal fluid via an intrathecal injection, allowing it to specifically target the central nervous system more effectively. Researchers are excited about Nusinersen because it offers a new approach to addressing the underlying cause of SMA, potentially leading to more significant and lasting improvements for patients.
What evidence suggests that Nusinersen might be an effective treatment for spinal muscular atrophy?
Research has shown that Nusinersen, which participants in this trial will receive, effectively treats spinal muscular atrophy (SMA). Studies have found that Nusinersen improves muscle strength and movement abilities in people with SMA. Evidence from earlier research also suggests that Nusinersen can delay or prevent the need for breathing support in babies with SMA. Long-term studies indicate that the treatment remains effective and safe over time. Overall, Nusinersen has proven to improve health outcomes for those with SMA.56789
Who Is on the Research Team?
Medical Director
Principal Investigator
Biogen
Are You a Good Fit for This Trial?
This trial is for infants aged ≤ 6 weeks with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). They must have a specific genetic makeup of SMA, an ulnar nerve test result ≥ 1 mV, and be born within certain gestational age ranges. Infants cannot participate if they've had previous treatments for SMA or show signs of low oxygen levels or abnormal blood tests.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive multiple doses of Nusinersen administered intrathecally
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