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Completed

NCT Number: NCT03878030

Effect of Nusinersen on Adults With Spinal Muscular Atrophy

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

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Key information

Conditions

Adult Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

18 year–60 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Northwell Health Neuroscience

Great Neck, New York, 11021, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

nusinersen

Drug

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

Other names: Spinraza

Primary outcomes

  1. Change in quantitative motor function

    Time frame: 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.

  2. Change in upper limb motor function

    Time frame: Two years

    Change from baseline to end of study in upper limb module score

  3. Change in 6 minute walk test in ambulatory patients

    Time frame: Two years

    Change in 6 minute walk test from baseline to end of study

Secondary outcomes

  1. Change in pulmonary function by spirometry forced vital capacity (FVC)

    Time frame: Two years

    Change in FVC from baseline to end of study

  2. Change in 10 meter walk test in ambulatory patients

    Time frame: Two years

    Change from baseline to end of study in the 10 meter walk test

  3. Change in compound muscle action potential (CMAP) amplitude by nerve conduction velocity (NCV) criteria

    Time frame: Two years

    Change from baseline to end of study in CMAP amplitude of responses from median, ulnar and peroneal motor nerves

  4. Change in pulmonary function by spirometry forced expiratory volume (FEV)

    Time frame: Two years

    Change from baseline to end of study in FEV

Sponsors and collaborators

Lead sponsor

Northwell Health

Other

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2021
Study completion
2021
First posted
Mar 18, 2019
Registry last updated
Mar 20, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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