NCT03878030

Effect of Nusinersen on Adults With Spinal Muscular Atrophy

Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 12 (actual)
• Sponsor: Northwell Health · Academic / Other
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

Observational study of adult patients with spinal muscular atrophy types 2 and 3 receiving nusinersen

Detailed description

Observational study to assess effects of nusinersen on motor function in adult patients with spinal muscular atrophy who are both ambulatory and non-ambulatory. Subjects will receive standard of care with nusinersen intrathecal injection and undergo baseline and every 6 month motor assessments and pulmonary function testing during the first two years of treatment with nusinersen.

Conditions

• Adult Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	nusinersen	Subjects will receive nusinersen and be observed with motor assessments for 24 months

Timeline

• Start date: 2019-04-01
• Primary completion: 2021-07-13
• Completion: 2021-07-13
• First posted: 2019-03-18
• Last updated: 2023-03-20

Locations

1 site across 1 country: United States