An observational study in Gaucher Disease, sponsored by John Bernat. Active, not recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-07-22.
Sponsored by John Bernat · Observational
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.
171 studies on the registry are indexed under Gaucher Disease; 37 are open to participants now.
This study's enrollment of 2 is below the median of 60 across 63 observational studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →This is the only study on the registry with John Bernat as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Subjects with Gaucher disease who previously received the investigational gene therapy product AVR-RD-02
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:
Subjects who received AVR-RD-02 in a separate parent trial
Biological: AVR-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.
Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder
Time frame: Baseline to Year 14
Change from baseline over time in hemoglobin concentration
Time frame: Baseline to Year 14
Change from baseline over time in platelet count
Time frame: Baseline to Year 14
Change from baseline over time in glucocerebrosidase (GCase) enzyme activity in whole blood
Time frame: Baseline to Year 14
Average vector copy number (VCN) from whole blood using quantitative polymerase chain reaction (qPCR) analysis
Time frame: Baseline to Year 14
Change from baseline over time in anti-GCase total antibodies and subsequent titers
Time frame: Baseline to Year 14
Identification of potential aberrant clonal expansion(s) assessed by performing insertional site analysis (ISA) from whole blood
Time frame: Baseline to Year 14
Presence of replication competent lentivirus (RCL)
Time frame: Baseline to Year 14
This study is active, not recruiting, as verified in Jul 2025. You cannot join it, but the record below documents what was studied.
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