Stopped: Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
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Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame
Timeframe: From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12
Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame
Timeframe: From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months)
Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment
Timeframe: Baseline, Day 30, 180, Month 12
Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment
Timeframe: Baseline, Day 30, Day 180, Month 12