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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 stratified in two groups, those < 24 months of age at time of dosing and those ≥ 24 months of age at time of dosing. This study will be conducted in 2 stages:
Stage 1: dose escalting study in children <24 months of age. Stage 2: the selecetd dose from Stage 1 in children ≥ 24 months of age.
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.
For patients enrolled from overseas, follow-up visits with patient's own paediatrician together with remote virtual visits are allowed.
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20 participants in 3 patient groups
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Central trial contact
Austin Gao, PhD
Data sourced from clinicaltrials.gov
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