Effect of Nusinersen on Adults With Spinal Muscular Atrophy

Northwell Health

Status: Completed

Conditions: Adult Spinal Muscular Atrophy

Treatments: Drug: nusinersen

Study type: Observational

Funder types: Other

Identifiers

NCT03878030

18-0149

Details and patient eligibility

About

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

Full 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.

Enrollment: 12 patients

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Genetically confirmed 5q SMA
• ability to access intrathecal space for nusinersen injection

Exclusion criteria

• Renal impairment
• thrombocytopenia
• inability to access intrathecal space by CT or flouro guided injection

Trial design

12 participants in 1 patient group

Subjects with spinal muscular atrophy types 2 and 3

Description:

Intrathecal nusinersen will be administered to all subjects per FDA approved label.

Treatment:

Drug: nusinersen