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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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22 August 2022 |
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Main ID: |
NCT03306277 |
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Date of registration:
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02/10/2017 |
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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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Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
STR1VE |
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Scientific title:
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Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion |
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Date of first enrolment:
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October 24, 2017 |
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Target sample size:
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22 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT03306277 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 3
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