A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- AVROBIO
- 入组人数
- 4
- 试验地点
- 3
- 主要终点
- Change from Baseline over time in platelet count
研究概览
简要总结
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).
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-02 treatment study.
结局指标
主要结局
Change from Baseline over time in platelet count
时间窗: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by physical examinations findings
时间窗: Baseline to Year 15 post gene therapy infusion
Change from baseline over time in spleen volume as assessed by abdominal Magnetic Resonance Imaging (MRI)
时间窗: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities as assessed by vital signs
时间窗: Baseline to Year 15 post gene therapy infusion
Baseline over time in liver volume as assessed by abdominal MRI
时间窗: Baseline to Year 15 post gene therapy infusion
Baseline over time in hemoglobin
时间窗: 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)
时间窗: Baseline to Year 15 post gene therapy infusion
Incidence of newly-diagnosed malignancy, hematologic disorder, and/or immune related events/immunogenicity
时间窗: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests
时间窗: Baseline to Year 15 post gene therapy infusion
Number of participants with clinically relevant abnormalities, as assessed by electrocardiograms (ECGs)
时间窗: Baseline to Year 15 post gene therapy infusion
次要结局
- 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)