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).
Subjects who received AVR-RD-02 in the preceding treatment study and who meet all eligibility criteria may participate in the AVRO-RD-02-LTF01 study. For individual participating subjects, the Baseline visit for the AVRO-RD-02-LTF01 study will coincide with the subject's last visit in the preceding treatment study, whenever feasible. Subjects will be asked to return for study visits at approximately 6-month intervals for the first 4 years of the study and then annually thereafter for 10 years (for a total follow-up of 14 years in this study), during which time periodic safety and efficacy assessments will be performed to assess measures of safety, engraftment, and clinical response after AVR-RD-02 treatment.
Study Type
OBSERVATIONAL
Enrollment
4
Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention
University of Iowa
Iowa City, Iowa, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
M.A.G.I.C. Clinic Ltd
Calgary, Calgary Alberta, Canada
Change from baseline over time in spleen volume as assessed by abdominal Magnetic Resonance Imaging (MRI)
Time frame: Baseline to Year 15 post gene therapy infusion
Baseline over time in liver volume as assessed by abdominal MRI
Time frame: Baseline to Year 15 post gene therapy infusion
Baseline over time in hemoglobin
Time frame: Baseline to Year 15 post gene therapy infusion
Change from Baseline over time in platelet count
Time frame: 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)
Time frame: Baseline to Year 15 post gene therapy infusion
Incidence of newly-diagnosed malignancy, hematologic disorder, and/or immune related events/immunogenicity
Time frame: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities as assessed by vital signs
Time frame: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by physical examinations findings
Time frame: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests
Time frame: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by electrocardiograms (ECGs)
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Time frame: Baseline to Year 15 post gene therapy infusion
Change from baseline over time in glucocerebrosidase (GCase) GCase enzyme activity level in
Time frame: Baseline to Year 15 post gene therapy infusion
Change from baseline over time in Enzyme Replacement Therapy (ERT) frequency and dosing
Time frame: Baseline to Year 15 post gene therapy infusion
Changes in Gaucher biomarker indices of Gaucher disease in chitotriosidase enzyme activity levels in plasma
Time frame: Baseline to Year 15 post gene therapy infusion
Changes in Gaucher biomarker indices of Gaucher disease in bone marrow burden (BMB) score as assessed by bone MRI
Time frame: Baseline to Year 15 post gene therapy infusion
Changes in Gaucher biomarker indices of Gaucher disease in bone mineral density (BMD) as assessed by dual-energy X-ray absorptiometry (DXA)
Time frame: Baseline to Year 15 post gene therapy infusion
Average Vector Copy Number (VCN) in bone marrow aspirate as assessed by quantitative polymerase chain reaction (qPCR) and/or droplet digital polymerase chain reaction (ddPCR)
Time frame: Baseline to Year 15 post gene therapy infusion
Change from Baseline in anti-GCase total antibodies and subsequent titers by an electrochemiluminescence method
Time frame: Baseline to Year 15 post gene therapy infusion
Changes in Gaucher disease in potential aberrant clonal expansion(s) assessed by performing insertional site analysis (ISA)
Time frame: Baseline to Year 15 post gene therapy infusion
Presence of replication competent lentivirus (RCL)
Time frame: Baseline to Year 15 post gene therapy infusion