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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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11 March 2024 |
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Main ID: |
NCT03421977 |
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Date of registration:
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30/01/2018 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Long-Term Follow-up Study for Patients From AVXS-101-CL-101
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Scientific title:
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A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101 |
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Date of first enrolment:
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September 21, 2017 |
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Target sample size:
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13 |
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Recruitment status: |
Active, not recruiting |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT03421977 |
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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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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Patient who received onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene
replacement therapy clinical trial for SMA Type 1.
2. Parent/legal guardian willing and able to complete the informed consent process,
comply with study procedures and visit schedule.
Exclusion Criteria:
1. Parent/legal guardian unable or unwilling to participate in the long term follow up
safety study.
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Spinal Muscular Atrophy 1
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Intervention(s)
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Biological: Onasemnogene Abeparvovec-xioi
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Primary Outcome(s)
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Long-Term Safety
[Time Frame: 15 years]
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Secondary ID(s)
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COAV101A12401
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AVXS-101-LT-001
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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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