Stopped: This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety or efficacy issue
This is a multicenter, multinational long-term follow-up study to assess the long-term safety and durability of effect of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).
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Change from baseline over time in spleen volume as assessed by abdominal Magnetic Resonance Imaging (MRI)
Timeframe: Baseline to Year 15 post gene therapy infusion
Baseline over time in liver volume as assessed by abdominal MRI
Timeframe: Baseline to Year 15 post gene therapy infusion
Baseline over time in hemoglobin
Timeframe: Baseline to Year 15 post gene therapy infusion
Change from Baseline over time in platelet count
Timeframe: Baseline to Year 15 post gene therapy infusion
Change from Baseline in plasma lyso-Gb1 levels by liquid chromatography tandem mass spectrometry (LC/MS/MS)
Timeframe: Baseline to Year 15 post gene therapy infusion
Incidence of newly-diagnosed malignancy, hematologic disorder, and/or immune related events/immunogenicity
Timeframe: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities as assessed by vital signs
Timeframe: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by physical examinations findings
Timeframe: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests
Timeframe: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by electrocardiograms (ECGs)
Timeframe: Baseline to Year 15 post gene therapy infusion