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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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27 March 2023 |
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Main ID: |
NCT03878030 |
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Date of registration:
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10/03/2019 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Effect of Nusinersen on Adults With Spinal Muscular Atrophy
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Scientific title:
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Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3 |
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Date of first enrolment:
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April 1, 2019 |
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Target sample size:
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12 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT03878030 |
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Study type:
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Observational |
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Study design:
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Phase:
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Countries of recruitment
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United States
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Contacts
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Name:
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Anthony Geraci, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Northwell Health |
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Key inclusion & exclusion criteria
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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
Age minimum:
18 Years
Age maximum:
60 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Adult Spinal Muscular Atrophy
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Intervention(s)
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Drug: nusinersen
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Primary Outcome(s)
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Change in upper limb motor function
[Time Frame: Two years]
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Change in 6 minute walk test in ambulatory patients
[Time Frame: Two years]
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Change in quantitative motor function
[Time Frame: Two years]
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Secondary Outcome(s)
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Change in 10 meter walk test in ambulatory patients
[Time Frame: Two years]
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Change in pulmonary function by spirometry forced vital capacity (FVC)
[Time Frame: Two years]
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Change in compound muscle action potential (CMAP) amplitude by nerve conduction velocity (NCV) criteria
[Time Frame: Two years]
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Change in pulmonary function by spirometry forced expiratory volume (FEV)
[Time Frame: Two years]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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