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Effect of Nusinersen on Adults With Spinal Muscular Atrophy

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ClinicalTrials.gov Identifier: NCT03878030
Recruitment Status : Recruiting
First Posted : March 18, 2019
Last Update Posted : May 30, 2019
Sponsor:
Information provided by (Responsible Party):
Anthony Geraci, Northwell Health

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

Condition or disease Intervention/treatment
Adult Spinal Muscular Atrophy Drug: 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.

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Study Type : Observational
Estimated Enrollment : 40 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 : April 1, 2019
Estimated Primary Completion Date : January 1, 2023
Estimated Study Completion Date : January 1, 2023


Group/Cohort 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 Name: Spinraza




Primary Outcome Measures :
  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 Outcome Measures :
  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



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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03878030


Contacts
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Contact: Anthony Geraci, MD 516-325-7000 ageraci@northwell.edu
Contact: Connie Lau 516-325-7000 clau@northwell.edu

Locations
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United States, New York
Northwell Health Neuroscience Recruiting
Great Neck, New York, United States, 11021
Contact: Anthony Geraci, MD    516-325-7000    ageraci@northwell.edu   
Contact: Connie Lau    516-325-7000    clau@northwell.edu   
Sponsors and Collaborators
Northwell Health
Investigators
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Principal Investigator: Anthony Geraci, MD Northwell Health

Publications of Results:
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Responsible Party: Anthony Geraci, Director, Neuromuscular medicine, Northwell Health
ClinicalTrials.gov Identifier: NCT03878030     History of Changes
Other Study ID Numbers: 18-0149
First Posted: March 18, 2019    Key Record Dates
Last Update Posted: May 30, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Atrophy
Muscular Atrophy
Muscular Atrophy, Spinal
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases