Gaucher Disease
Conditions
Brief summary
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).
Detailed description
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.
Interventions
BIOLOGICALAVR-RD-02
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.
Sponsors
John Bernat
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Subjects must meet all of the following inclusion criteria for participation in this study: 1. Subject must be willing and able to provide written informed consent for the JAB-GD-001 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures. 2. Subject must have been enrolled in the preceding AVROBIO AVRO-RD-02-201 treatment study and have received AVR-RD-02 treatment
Exclusion criteria
Subject has any medical, psychological, or other condition that, in the opinion of the Investigator: * Might interfere with the subject's participation in the study (including consenting to procedures); and/or * Poses any additional risk to the subject; and/or * Might confound the results of any study-required assessments.
Design outcomes
Primary
| Measure | 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 |
Secondary
| Measure | Time frame |
|---|---|
| Identification of potential aberrant clonal expansion(s) assessed by performing insertional site analysis (ISA) from whole blood | Baseline to Year 14 |
| Change from baseline over time in glucocerebrosidase (GCase) enzyme activity in whole blood | Baseline to Year 14 |
| Presence of replication competent lentivirus (RCL) | Baseline to Year 14 |
| Average vector copy number (VCN) from whole blood using quantitative polymerase chain reaction (qPCR) analysis | Baseline to Year 14 |
| Change from baseline over time in anti-GCase total antibodies and subsequent titers | Baseline to Year 14 |
Countries
United States
Outcome results
None listed