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Effect of Nusinersen on Adults With Spinal Muscular Atrophy

Sponsor
Northwell Health (Other)
Overall Status
Active, not recruiting
CT.gov ID
NCT03878030
Collaborator
(none)
12
Enrollment
1
Location
45
Anticipated Duration (Months)
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase

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.

Study Design

Study Type:
Observational
Anticipated Enrollment :
12 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3
Actual Study Start Date :
Apr 1, 2019
Anticipated Primary Completion Date :
Jan 1, 2023
Anticipated Study Completion Date :
Jan 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Subjects with spinal muscular atrophy types 2 and 3

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

Drug: nusinersen
Subjects will receive nusinersen and be observed with motor assessments for 24 months
Other Names:
  • Spinraza

    		•

    Outcome Measures

    Primary Outcome Measures

    1. 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.

    2. Change in upper limb motor function [Two years]

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

    3. Change in 6 minute walk test in ambulatory patients [Two years]

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

    Secondary Outcome Measures

    1. Change in pulmonary function by spirometry forced vital capacity (FVC) [Two years]

      Change in FVC from baseline to end of study

    2. Change in 10 meter walk test in ambulatory patients [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 [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) [Two years]

      Change from baseline to end of study in FEV

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 60 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Northwell Health Neuroscience Great Neck New York United States 11021

    Sponsors and Collaborators

    • Northwell Health

    Investigators

    • Principal Investigator: Anthony Geraci, MD, Northwell Health

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Anthony Geraci, Director, Neuromuscular medicine, Northwell Health
    ClinicalTrials.gov Identifier:
    NCT03878030
    Other Study ID Numbers:
    • 18-0149
    First Posted:
    Mar 18, 2019
    Last Update Posted:
    Dec 1, 2021
    Last Verified:
    Nov 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Muscular Atrophy
    Muscular Atrophy, Spinal
    Atrophy

    Study Results

    No Results Posted as of Dec 1, 2021