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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 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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Subjects with Gaucher 1 Disease | This is a long-term follow-up study of subjects who previously received AVR-RD-02 (single dose administration) in a preceding 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 |
|---|---|---|
| Change from baseline over time in spleen volume as assessed by abdominal Magnetic Resonance Imaging (MRI) | Baseline to Year 15 post gene therapy infusion | |
| Baseline over time in liver volume as assessed by abdominal MRI | Baseline to Year 15 post gene therapy infusion | |
| Baseline over time in hemoglobin | Baseline to Year 15 post gene therapy infusion | |
| Change from Baseline over time in platelet count | 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) | Baseline to Year 15 post gene therapy infusion | |
| Incidence of newly-diagnosed malignancy, hematologic disorder, and/or immune related events/immunogenicity | 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 | |
| Number of participants with clinically relevant abnormalities, as assessed by physical examinations findings | Baseline to Year 15 post gene therapy infusion | |
| Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline over time in glucocerebrosidase (GCase) GCase enzyme activity level in | Baseline to Year 15 post gene therapy infusion | |
| Change from baseline over time in Enzyme Replacement Therapy (ERT) frequency and dosing | Baseline to Year 15 post gene therapy infusion |
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Inclusion Criteria:
Exclusion Criteria:
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To permit longitudinal assessment of safety, efficacy and durability of effect of AVR-RD-02 gene therapy infusion in the AVRO-RD-02-LTF01 study, subjects must have received AVR-RD-02 in a preceding treatment study and agree to comply with the study visit schedule and procedures for AVRO-RD-02-LTF01 for inclusion in the Long-Term Follow-Up study.
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| Name | Affiliation | Role |
|---|---|---|
| Milena Veselinovic, MD | AVROBIO | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Iowa | Iowa City | Iowa | 52242 | United States | ||
| Hackensack University Medical Center |
To keep consistent with the current treatment listing
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| Type | Date | Date Unknown |
|---|---|---|
| Release | Nov 9, 2023 | |
| Unrelease | Jan 8, 2024 | |
| Release | Jan 8, 2024 | |
| Reset | Jul 3, 2024 |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Nov 9, 2023 | Jan 8, 2024 | |||
| Jan 8, 2024 |
| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| 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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| Baseline to Year 15 post gene therapy infusion |
| Number of participants with clinically relevant abnormalities, as assessed by electrocardiograms (ECGs) | Baseline to Year 15 post gene therapy infusion |
| Changes in Gaucher biomarker indices of Gaucher disease in chitotriosidase enzyme activity levels in plasma | 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 | 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) | 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) | Baseline to Year 15 post gene therapy infusion |
| Change from Baseline in anti-GCase total antibodies and subsequent titers by an electrochemiluminescence method | 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) | Baseline to Year 15 post gene therapy infusion |
| Presence of replication competent lentivirus (RCL) | Baseline to Year 15 post gene therapy infusion |
| Hackensack |
| New Jersey |
| 07601 |
| United States |
| M.A.G.I.C. Clinic Ltd | Calgary | Calgary Alberta | T2M 0L6 | Canada |
| Jul 3, 2024 |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |