Status and phase
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About
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
Full description
Study duration per participant is approximately 25 months including an approximately 30-day screening/baseline period, an approximately 24-month study observation period including 1 treatment day, and an approximately 24-month follow-up period.
Patients will be tested at baseline and return for follow-up visits twice a week through the first month post dose, and followed by visits at months 2, 3, 6 12, 18 and 24 post infusion. Unscheduled visits may occur if the investigator determines that they are necessary.
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Inclusion criteria
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Allocation
Interventional model
Masking
6 participants in 1 patient group
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Central trial contact
Austin Gao, PhD
Data sourced from clinicaltrials.gov
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