Long Term Follow-Up for Safety of AVR-RD-02
Active, not recruiting
- Conditions
- Gaucher Disease
- Interventions
- Biological: AVR-RD-02
- Registration Number
- NCT06488261
- Lead Sponsor
- John Bernat
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 2
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Subjects who received AVR-RD-02 AVR-RD-02 Subjects who received AVR-RD-02 in a separate parent trial
- Primary Outcome Measures
Name Time Method Chane from baseline over time in platelet count Baseline to Year 14 Change from baseline over time in hemoglobin concentration Baseline to Year 14 Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder Baseline to Year 14
- Secondary Outcome Measures
Name Time Method 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 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 Change from baseline over time in anti-GCase total antibodies and subsequent titers Baseline to Year 14
Trial Locations
- Locations (1)
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States