Evaluating the safety and effectiveness of Spinraza injection for spinal muscular atrophy
A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea
This study is testing how safe and effective the Spinraza injection is for people with spinal muscular atrophy in Korea who are starting or already receiving this treatment.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 145 (estimated) |
| Sex | All |
| Sponsor | Biogen Industry-sponsored |
| Locations | 16 sites (Busan and 15 other locations) |
| Trial ID | NCT04317794 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the safety and effectiveness of nusinersen sodium injection, known as Spinraza, in patients with spinal muscular atrophy (SMA) in Korea. Participants will be those currently receiving or about to start treatment with Spinraza, and the study will monitor their responses in a real-world setting. The study will collect data on adverse effects and treatment outcomes to provide insights into the drug's performance outside of controlled clinical trials.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with 5q-linked spinal muscular atrophy who are currently receiving or about to initiate treatment with Spinraza.
Not a fit: Patients who are hypersensitive to Spinraza or are currently participating in other interventional clinical trials for SMA may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable information on the long-term safety and effectiveness of Spinraza, improving treatment strategies for patients with SMA.
How similar studies have performed: While this study is observational and focuses on postmarketing surveillance, similar studies have previously shown the effectiveness of nusinersen in controlled settings, suggesting potential for success in this real-world evaluation.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: * Currently receiving or about to initiate treatment with commercial Spinraza in the postmarketing setting * Genetic documentation of 5q-linked SMA Key Exclusion Criteria: * Hypersensitivity to the active substance or any of the excipients of Spinraza * Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA * Inability to comply with study requirements NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Where this trial is running
Busan and 15 other locations
- Pusan Natioanl University Hospital — Busan, Korea, Republic of (Completed)
- Samsung Changwon Hospital — ChangWon, Korea, Republic of (Recruiting)
- Chungbuk National University Hospital — Cheongju-si, Korea, Republic of (Recruiting)
- Kyungpook National University Hospital — Daegu, Korea, Republic of (Recruiting)
- Yeungnam University Hospital — Daegu, Korea, Republic of (Recruiting)
- Chungnam National University Hospital — Daejeon, Korea, Republic of (Recruiting)
- Chonnam National University Hospital — Gwangju, Korea, Republic of (Recruiting)
- Chonbuk National University Hospital — Jeonju-si, Korea, Republic of (Recruiting)
- Severance Hospital — Seoul, Korea, Republic of (Recruiting)
- Asan Medical Center — Seoul, Korea, Republic of (Recruiting)
- Korea university Anam Hospital — Seoul, Korea, Republic of (Recruiting)
- Korea University Guro Hospital — Seoul, Korea, Republic of (Recruiting)
- Samsung Medical Center — Seoul, Korea, Republic of (Recruiting)
- Seoul Natioanl University Hospital — Seoul, Korea, Republic of (Recruiting)
- Severance Hospital, Yonsei University Health System — Seoul, Korea, Republic of (Recruiting)
- Pusan National University Yangsan Hospital — Yangsan, Korea, Republic of (Recruiting)
Study contacts
- Study coordinator: US Biogen Clinical Trial Center
- Email: clinicaltrials@biogen.com
- Phone: 866-633-4636
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.