This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-01 treatment in participants who received a single dose administration of lentiviral gene therapy in Study AVRO-RD-01-201 (treatment study). No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from AVR-RD-01 gene therapy infusion.
Participants enrolled in the AVRO-RD-01-201 study will be offered participation in the AVRO-RD-01-LTF01 study. The Baseline visit for the AVRO-RD-01-LTF01 study will coincide with the participant's last visit in the AVRO-RD-01-201 study. Participants confirmed eligible for the AVRO-RD-01-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for 10 years (for a total of 15 years from AVR-RD-01 infusion) during which time continued safety, engraftment, and efficacy of treatment will be assessed.
Study Type
OBSERVATIONAL
Enrollment
5
Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.
Royal Melbourne Hospital
Melbourne, Parkville VIC, Australia
Royal Perth Hospital
Perth, Australia
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil
Incidence of clinically significant AEs and SAEs
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 vital signs
Time frame: Baseline to Year 15 post gene therapy infusion
Presence of anti-Alpha-galactosidase A (AGA) antibodies
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
Evaluate for the presence of aberrant clonal expansion as assessed by integration site analysis (ISA)
Time frame: Baseline to Year 15 post gene therapy infusion
Change from baseline in AGA enzyme activity level and peripheral blood leukocytes (PBLs)
Time frame: Baseline to Year 15 post gene therapy infusion
Average Vector Copy Number (VCN) in peripheral blood leukocytes 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 Globotriaosylceramide (Gb3) biomarkers for Fabry disease in plasma and urine
Time frame: Baseline to Year 15 post gene therapy infusion
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Average Vector Copy Number (VCN) in bone marrow / progenitor cells 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 eGFR
Time frame: Baseline to Year 15 post gene therapy infusion
Change from baseline in left ventricular mass index (LVMI) as assessed by cardiac magnetic resonance imaging (MRI)
Time frame: Baseline to Year 15 post gene therapy infusion
Change from baseline in abdominal pain and stool consistency as assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D)
Time frame: Baseline to Year 15 post gene therapy infusion
Change from baseline in Brief Pain Inventory-Short Form (BPI-SF) questionnaire scores
Time frame: Baseline to Year 15 post gene therapy infusion
Change from baseline in physical and mental functioning as assessed by the Short Form 36 (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS) scores
Time frame: Baseline to Year 15 post gene therapy infusion