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Purpose of Study:
This study is a phase I, single-site, dose escalation study to evaluate the safety and efficacy of gene transfer for Spinal Muscular Atrophy Type 1 (SMA1). Enrollment is at Nationwide Children’s Hospital in Columbus, Ohio. Infants between 0 and 9 months of age with SMA1 at the time of gene transfer may be eligible to take part in this first human trial. A total of nine patients will be enrolled to receive a one-time gene transfer infusion. Patients will continue to be monitored at Nationwide Children’s Hospital including physical exams and blood tests for two years after gene transfer.
Who can participate in this study:
Age: Nine months of age and younger at the time of gene transfer
*Patients may be put on non-invasive ventilator support (BiPAP) for less than 16 hours a day at the discretion of their physician or research staff.
Who to contact:
If you are interested in this study, please contact the Resarech Cocordinator, Sohyun McElroy, Ph.D., CCRC, Phone: (614) 355-2606.
A description of the study is also listed at the website www.ClinicalTrials.gov with the identifier NCT02122952