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This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety or efficacy issue.
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Participants with Fabry Disease | This is a long-term follow-up study of participants who previously received AVR-RD-01 (single dose administration) in the AVRO-RD-01-201 treatment study. No investigational product will be administered in this study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Safety and Efficacy Assessments | Other | Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of clinically significant AEs and SAEs | Baseline to Year 15 post gene therapy infusion | |
| Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests | Baseline to Year 15 post gene therapy infusion | |
| Number of participants with clinically relevant abnormalities, as assessed by vital signs | Baseline to Year 15 post gene therapy infusion | |
| Presence of anti-Alpha-galactosidase A (AGA) antibodies | Baseline to Year 15 post gene therapy infusion | |
| Presence of replication competent lentivirus (RCL) | Baseline to Year 15 post gene therapy infusion | |
| Evaluate for the presence of aberrant clonal expansion as assessed by integration site analysis (ISA) | Baseline to Year 15 post gene therapy infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in AGA enzyme activity level and peripheral blood leukocytes (PBLs) | 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) |
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Inclusion Criteria:
1. Subject must have been enrolled and received AVR-RD-01 in the AVRO-RD-01-201 study.
Exclusion Criteria:
Subject has any medical, psychological, or other condition that, in the opinion of the Investigator:
Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-01 treatment study.
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Subjects who received AVR-RD-01 in the preceding treatment study, meet all eligibility criteria, and agree to comply with the study visit schedule and procedures.
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| Name | Affiliation | Role |
|---|---|---|
| Inderpal Panesar, MRPharmS | AVROBIO, Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Royal Melbourne Hospital | Melbourne | Parkville VIC | Australia | |||
| Royal Perth Hospital |
| Type | Date | Date Unknown |
|---|---|---|
| Release | Nov 9, 2023 | |
| Unrelease | Jan 8, 2024 | |
| Release | Jan 12, 2024 | |
| Reset | Jul 11, 2024 |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Nov 9, 2023 | Jan 8, 2024 | |||
| Jan 12, 2024 |
| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| D016464 | Lysosomal Storage Diseases |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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| ID | Term |
|---|---|
| D012449 | Safety |
| ID | Term |
|---|---|
| D000056 | Accident Prevention |
| D000059 | Accidents |
| D011634 | Public Health |
| D004778 | Environment and Public Health |
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Blood sample collections including serum chemistry (and electrolytes), hematology, and anti-AGA antibodies, and urinalysis. Bone marrow aspirate for efficacy, and reproductive testing.
|
| Baseline to Year 15 post gene therapy infusion |
| Change from baseline in Globotriaosylceramide (Gb3) biomarkers for Fabry disease in plasma and urine | Baseline to Year 15 post gene therapy infusion |
| 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) | Baseline to Year 15 post gene therapy infusion |
| Change from baseline in eGFR | 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) | 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) | Baseline to Year 15 post gene therapy infusion |
| Change from baseline in Brief Pain Inventory-Short Form (BPI-SF) questionnaire scores | 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 | Baseline to Year 15 post gene therapy infusion |
| Perth |
| Australia |
| Hospital de Clinicas de Porto Alegre | Porto Alegre | Rio Grande do Sul | 90420-020 | Brazil |
| Jul 11, 2024 |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |