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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03878030

Enrollment

Registered

2019-03-18

Start date

2019-04-01

Completion date

2021-07-13

Last updated

2023-03-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Adult Spinal Muscular Atrophy

Brief 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.

Interventions

DRUGnusinersen

Subjects will receive nusinersen and be observed with motor assessments for 24 months

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to 60 Years

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

Design outcomes

Primary

Measure	Time frame	Description
Change in quantitative motor function	Two years	Primary Endpoint: Change from baseline to end of study in quantitative motor strength by dynamometry of upper limb muscles - shoulder abduction and elbow flexion/extension.
Change in upper limb motor function	Two years	Change from baseline to end of study in upper limb module score
Change in 6 minute walk test in ambulatory patients	Two years	Change in 6 minute walk test from baseline to end of study

Secondary

Measure	Time frame	Description
Change in pulmonary function by spirometry forced vital capacity (FVC)	Two years	Change in FVC from baseline to end of study
Change in pulmonary function by spirometry forced expiratory volume (FEV)	Two years	Change from baseline to end of study in FEV
Change in 10 meter walk test in ambulatory patients	Two years	Change from baseline to end of study in the 10 meter walk test
Change in compound muscle action potential (CMAP) amplitude by nerve conduction velocity (NCV) criteria	Two years	Change from baseline to end of study in CMAP amplitude of responses from median, ulnar and peroneal motor nerves

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026