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Effect of Nusinersen on Adults With Spinal Muscular Atrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03878030
Recruitment Status : Active, not recruiting
First Posted : March 18, 2019
Last Update Posted : February 28, 2020
Sponsor:
Information provided by (Responsible Party):
Anthony Geraci, Northwell Health

Tracking Information
First Submitted Date March 10, 2019
First Posted Date March 18, 2019
Last Update Posted Date February 28, 2020
Actual Study Start Date April 1, 2019
Estimated Primary Completion Date January 1, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: March 14, 2019)
  • 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.
  • Change in upper limb motor function [ Time Frame: Two years ]
    Change from baseline to end of study in upper limb module score
  • 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
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: March 14, 2019)
  • Change in pulmonary function by spirometry forced vital capacity (FVC) [ Time Frame: Two years ]
    Change in FVC from baseline to end of study
  • 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
  • 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
  • Change in pulmonary function by spirometry forced expiratory volume (FEV) [ Time Frame: Two years ]
    Change from baseline to end of study in FEV
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Effect of Nusinersen on Adults With Spinal Muscular Atrophy
Official Title Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3
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.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population The population to be studied are a homogenous group of adult subjects with 5q SMA seeking treatment with nusinersen. The subjects will have a broad phenotype spectrum of motor weakness including ability to ambulate, non-ambulatory and varying degrees of upper limb motor function and respiratory and swallow abilities, with some more affected and requiring invasive ventilation or NIV, and/or PEG tubes for nutrition.
Condition Adult Spinal Muscular Atrophy
Intervention Drug: nusinersen
Subjects will receive nusinersen and be observed with motor assessments for 24 months
Other Name: Spinraza
Study Groups/Cohorts Subjects with spinal muscular atrophy types 2 and 3
Intrathecal nusinersen will be administered to all subjects per FDA approved label.
Intervention: Drug: nusinersen
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Estimated Enrollment
 (submitted: February 26, 2020)
12
Original Estimated Enrollment
 (submitted: March 14, 2019)
40
Estimated Study Completion Date January 1, 2023
Estimated Primary Completion Date January 1, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria

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
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03878030
Other Study ID Numbers 18-0149
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Anthony Geraci, Northwell Health
Study Sponsor Northwell Health
Collaborators Not Provided
Investigators
Principal Investigator: Anthony Geraci, MD Northwell Health
PRS Account Northwell Health
Verification Date February 2020