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).
Age range
18 Years – 99 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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