NCT03709784

Spinraza in Adult Spinal Muscular Atrophy

A Prospective, Multi-center, Observational Study of the Safety, Tolerability and Effectiveness of SPINRAZA® (Nusinersen) in Adult Patients With Spinal Muscular Atrophy

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 148 (estimated)
• Sponsor: Washington University School of Medicine · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This is a longitudinal, observational study of adult patients with genetically confirmed chromosome 5q SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.

Detailed description

This is a prospective, longitudinal, multi-center, observational study designed to evaluate the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) in ambulatory and non-ambulatory adult patients with SMA. Subjects with SMA II/III that are 18 years to 70 years of age who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan will be enrolled in this study. This study does not provide SPINRAZA® (nusinersen) or cover costs associated with standard clinical care.These patients will be treated by their respective physicians according to standard clinical practice. Study visits, some of which including standardized assessments of strength and function, will occur at baseline, day 15 after treatment initiation, day 30, day 60, and then 4-month intervals through month 30. After 30 months an additional cohort 2 was added. The approval date was March 13, 2023. The cohort 2 is a one time survey to gain a better understanding of this adult population and their treatment preferences.

Conditions

• Spinal Muscular Atrophy
• Spinal Muscular Atrophy Type II
• Spinal Muscular Atrophy Type 3

Interventions

Type	Name	Description
DRUG	Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care	This is an observational study of adult patients with SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.
OTHER	One time survey	One time survey

Timeline

• Start date: 2018-08-16
• Primary completion: 2024-12-31
• Completion: 2025-01-01
• First posted: 2018-10-17
• Last updated: 2024-11-20

Locations

11 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study