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The goal of this observational study is to assess the safety and tolerability 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 (NCT04145037) and who meet all eligibility criteria may participate. Subjects will be asked to return for study visits annually for 14 years to assess measures of safety and persistence of AVR-RD-02 treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Subjects who received AVR-RD-02 | Subjects who received AVR-RD-02 in a separate parent trial |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AVR-RD-02 | Biological | No study drug is administered in this study. Subjects who received AVR-RD-02 in a separate parent trial will be evaluated in this trial for long-term safety. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder | Baseline to Year 14 | |
| Change from baseline over time in hemoglobin concentration | Baseline to Year 14 | |
| Change from baseline over time in platelet count | Baseline to Year 14 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline over time in glucocerebrosidase (GCase) enzyme activity in whole blood | Baseline to Year 14 | |
| Average vector copy number (VCN) from whole blood using quantitative polymerase chain reaction (qPCR) analysis | Baseline to Year 14 |
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Inclusion Criteria
Subjects must meet all of the following inclusion criteria for participation in this study:
Exclusion Criteria
Subject has any medical, psychological, or other condition that, in the opinion of the Investigator:
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Subjects with Gaucher disease who previously received the investigational gene therapy product AVR-RD-02
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| Name | Affiliation | Role |
|---|---|---|
| John A. Bernat, MD, PhD | University of Iowa | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States |
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| 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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| Change from baseline over time in anti-GCase total antibodies and subsequent titers | Baseline to Year 14 |
| Identification of potential aberrant clonal expansion(s) assessed by performing insertional site analysis (ISA) from whole blood | Baseline to Year 14 |
| Presence of replication competent lentivirus (RCL) | Baseline to Year 14 |
| 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 |