A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
- Conditions
- Type 1 Gaucher Disease
- Interventions
- Other: Safety and efficacy assessments
- Registration Number
- NCT04836377
- Lead Sponsor
- AVROBIO
- Brief Summary
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).
- Detailed Description
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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 4
- Subject must have been enrolled and received AVR-RD-02 as single dose administration
- Subject must be willing and able to provide written informed consent for the AVRO-RD-02-LTF01 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures.
- Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-02 treatment study.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Subjects with Gaucher 1 Disease Safety and efficacy assessments 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.
- Primary Outcome Measures
Name Time Method Change from Baseline over time in platelet count 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 Change from baseline over time in spleen volume as assessed by abdominal Magnetic Resonance Imaging (MRI) 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 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 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 clinical laboratory tests 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
- Secondary Outcome Measures
Name Time Method 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 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 Presence of replication competent lentivirus (RCL) 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 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
Trial Locations
- Locations (3)
Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
M.A.G.I.C. Clinic Ltd
🇨🇦Calgary, Calgary Alberta, Canada