NCT04591678

Adults With SMA Treated With Nusinersen

Characterizing Longitudinal Outcomes in Adults With SMA Treated With Nusinersen

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 15 (actual)
• Sponsor: Ohio State University · Academic / Other
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This is a single center, 22-month observational study of nusinersen treatment in adult patients with spinal muscular atrophy (SMA). There will be a total of seven visits. Nusinersen is provided as standard of care and not considered research in this study. Information will be collected regarding the general health, and function including muscle strength of, as well as any positive and/or adverse events experienced by the study participants.

Detailed description

This is a single center, 22-month observational study of nusinersen treatment in adult patients with SMA. There will be a total of five visits. All subjects will be evaluated at a screening visit no more than four weeks before starting their standard of care nusinersen treatment to determine their eligibility for participation. Eligible patients will complete their standard induction intrathecal nusinersen treatment on day 1, 15, 29 and 60 followed by maintenance doses every four months. Subjects will be reevaluated after completing the loading doses at 2 months followed by every four-month evaluations for the total duration of the treatment of 22 months. Currently, there is no data published on patients receiving this treatment over the age of 18. Without published data collected from research, it is difficult for adult patients to get approval from their insurance companies to receive this treatment. This study hopes to collect data, in order to make this treatment option easier for patients with SMA to access and in general learn how well nusinersen is working in adult patients with SMA overtime.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	nusinersen	SPINRAZA (nusinersen) is FDA approved to treat spinal muscular atrophy (SMA) through intrathecal injection.

Timeline

• Start date: 2018-10-01
• Primary completion: 2021-05-14
• Completion: 2021-06-25
• First posted: 2020-10-19
• Last updated: 2021-09-22

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study